Peptides
104 peptides with graded research outcomes.
For research and education only. Many peptides are not FDA-approved. Talk to a healthcare professional before using one.
Peptide list
- Best evidence:
MOTS-c
MOTS-c (Mitochondrial Open Reading Frame of the 12S rRNA Type-c) is a 16-amino-acid mitochondrial-derived peptide encoded within the 12S rRNA gene. Discovered in 2015, it regulates metabolic homeostasis, insulin sensitivity, and has been shown to mimic some beneficial effects of exercise. MOTS-c levels naturally decline with age, and the peptide is being studied for age-related metabolic diseases.
- Best evidence:
Liraglutide (Victoza/Saxenda)
Liraglutide is a GLP-1 receptor agonist peptide approved by the FDA for type 2 diabetes (Victoza, 1.8mg daily) and chronic weight management (Saxenda, 3.0mg daily). It is a 97% homologous analog of human GLP-1 with a fatty acid side chain enabling albumin binding and once-daily dosing. The LEAD, SCALE, and LEADER clinical trial programs demonstrated significant benefits for glycemic control, weight loss, and cardiovascular outcomes.
- Best evidence:
Cerebrolysin
Cerebrolysin is a neuropeptide preparation derived from porcine brain tissue, consisting of low-molecular-weight peptides and free amino acids that mimic the action of endogenous neurotrophic factors. It is approved in over 50 countries (though not in the USA) for the treatment of stroke, traumatic brain injury, and dementia. Multiple randomized controlled trials and meta-analyses have evaluated its efficacy, showing consistent improvements in early neurological recovery after ischemic stroke and modest cognitive benefits in Alzheimer's disease, with a safety profile comparable to placebo.
- Best evidence:
Dulaglutide (Trulicity)
Dulaglutide (Trulicity) is a GLP-1 receptor agonist peptide approved by the FDA for type 2 diabetes. It is a fusion protein consisting of two GLP-1 analogs covalently linked to a modified human IgG4-Fc heavy chain, enabling once-weekly dosing. The AWARD clinical trial program demonstrated efficacy for glycemic control, and the REWIND trial showed cardiovascular benefits in a broad population with T2DM.
- Best evidence:
Elamipretide (Forzinity/SS-31)
Elamipretide is a mitochondria-targeting tetrapeptide (D-Arg-Dmt-Lys-Phe-NH2) that binds cardiolipin to stabilize cristae structure and improve ATP production. FDA-approved September 2025 for Barth syndrome - the first FDA-approved therapy for any mitochondrial disease. TAZPOWER trial: 96m improvement in 6MWT (P=0.003), >45% improvement in knee extensor strength at 168 weeks. Also studied in heart failure (PROGRESS-HF) and STEMI (EMBRACE-STEMI) with mixed results.
- Best evidence:
Sermorelin
Sermorelin (GHRH 1-29) is a 29-amino-acid synthetic analog of growth hormone-releasing hormone (GHRH) that retains full biological activity. It was FDA-approved in 1990 for diagnostic use and in 1997 for treating childhood growth hormone deficiency. Sermorelin stimulates natural GH release from the pituitary gland through the hypothalamic-pituitary axis, producing physiological GH patterns with built-in safety through somatostatin feedback regulation.
- Best evidence:
Epitalon
Epitalon (Epithalamin) is a synthetic tetrapeptide (Ala-Glu-Asp-Gly) that activates telomerase, the enzyme responsible for maintaining telomere length. Developed from over 35 years of Russian research on pineal gland extracts, it may support cellular longevity and regulate melatonin production. Note: Epitalon is a research peptide not approved by the FDA for human use and is not available as a regulated pharmaceutical in most countries.
- Best evidence:
Exenatide (Byetta/Bydureon)
Exenatide is a GLP-1 receptor agonist peptide approved by the FDA for type 2 diabetes, available as twice-daily (Byetta) and once-weekly extended-release (Bydureon) formulations. It is a synthetic version of exendin-4, a 39-amino-acid peptide originally isolated from Gila monster saliva. The DURATION clinical trial program demonstrated efficacy for glycemic control and weight loss, while the EXSCEL trial assessed cardiovascular outcomes.
- Best evidence:
Semaglutide (Ozempic/Wegovy)
Semaglutide is a GLP-1 receptor agonist peptide approved by the FDA for type 2 diabetes (Ozempic, Rybelsus) and chronic weight management (Wegovy). It is a 31-amino-acid peptide analog of human GLP-1 with 94% structural similarity but modified for extended half-life (approximately 1 week). The STEP, SUSTAIN, and SELECT clinical trial programs demonstrated consistent, substantial benefits for glycemic control, weight loss, and cardiovascular outcomes.
- Best evidence:
TB-500
TB-500 is a synthetic fragment of Thymosin Beta-4, a naturally occurring 43-amino acid peptide present in most human cells. It has been investigated for its potential role in tissue repair, wound healing, and regeneration. Research, primarily in animal models, suggests it may promote angiogenesis, cell migration, and reduce inflammation. Human clinical trials are limited.
- Best evidence:
Tirzepatide (Mounjaro/Zepbound)
Tirzepatide is a first-in-class dual GIP/GLP-1 receptor agonist approved by the FDA for type 2 diabetes (Mounjaro) and chronic weight management (Zepbound). It is a 39-amino-acid synthetic peptide based on the native GIP sequence with a C20 fatty diacid modification enabling once-weekly dosing. The SURPASS and SURMOUNT clinical trial programs demonstrated unprecedented efficacy for glycemic control and weight loss, with up to 22.5% body weight reduction.
- Best evidence:
BPC-157
BPC-157 is a synthetic 15-amino-acid peptide derived from human gastric juice proteins. B-GRADE for tendon healing (6 studies, 180 participants) and gastric ulcer healing (8 studies, 240 participants). C-GRADE for ligament healing, muscle recovery, wound healing, and neuroprotection. CRITICAL CAVEAT: Nearly all evidence is from ANIMAL studies - human clinical trials are extremely limited. Popular in biohacking/sports communities but regulatory status unclear. NOT FDA-approved. Underground/gray market sourcing raises purity and safety concerns.
- Best evidence:
Tesamorelin
Tesamorelin is a 44-amino-acid synthetic analog of growth hormone-releasing hormone (GHRH) that is FDA-approved (2010) for reducing excess abdominal fat in HIV-infected patients with lipodystrophy. It stimulates endogenous pulsatile GH secretion from the pituitary gland. Tesamorelin has demonstrated efficacy in reducing visceral adipose tissue, liver fat (NAFLD), and improving cognitive function in clinical trials.
- Best evidence:
Bortezomib
Bortezomib is the first-in-class proteasome inhibitor for multiple myeloma and mantle cell lymphoma. FDA-approved 2003 (relapsed MM), 2008 (frontline), 2014 (MCL). A-GRADE evidence: APEX Phase 3 (n=669) showed 38% vs 18% response rate vs dexamethasone (P<0.001), 80% vs 66% 1-year survival. VISTA (n=682): VMP superior to MP in transplant-ineligible elderly. SAFETY CONCERN: Peripheral neuropathy 31-45% (dose-limiting), thrombocytopenia 26-30%. Subcutaneous route reduces neuropathy vs IV. Neuropathy 75-81% reversible with dose modification. PRESCRIPTION ONLY - oncology setting.
- Best evidence:
Calcitonin Salmon
Calcitonin salmon is a 32-amino acid peptide hormone analog used for postmenopausal osteoporosis and hypercalcemia. FDA-approved since 1975 as nasal spray (200 IU daily) and injection (100 IU daily). A-GRADE evidence: PROOF trial showed 33% reduction in vertebral fractures at 200 IU daily. A-GRADE for bone turnover marker reduction and acute fracture pain relief. B-GRADE for BMD increase and chronic bone pain. Has unique analgesic properties making it useful for acute osteoporotic fracture pain. Salmon calcitonin has 40-50x greater potency than human calcitonin. PRESCRIPTION ONLY - second-line osteoporosis treatment after bisphosphonates.
- Best evidence:
Denosumab (Prolia/Xgeva)
Denosumab is a fully human monoclonal antibody that inhibits RANKL (receptor activator of nuclear factor-κB ligand), the key mediator of osteoclast formation and activity. FDA-approved in 2010 for osteoporosis (Prolia, 60mg every 6 months) and bone metastases (Xgeva, 120mg monthly). The landmark FREEDOM trial demonstrated significant reductions in vertebral, hip, and nonvertebral fractures, with benefits sustained up to 10 years in extension studies.
- Best evidence:
Desmopressin (DDAVP)
Desmopressin (1-deamino-8-D-arginine vasopressin) is a synthetic analog of antidiuretic hormone (vasopressin). FDA-approved since 1978, it is used for central diabetes insipidus, nocturnal enuresis, hemophilia A, and von Willebrand disease. It has enhanced antidiuretic potency with minimal pressor effect and longer duration than native vasopressin.
- Best evidence:
Lanreotide (Somatuline)
Lanreotide is a long-acting synthetic octapeptide analog of somatostatin, FDA-approved for treatment of acromegaly and gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The landmark CLARINET trial demonstrated 53% reduction in disease progression risk in metastatic GEP-NETs. Available as an extended-release depot formulation (Somatuline Depot) for once-monthly injection.
- Best evidence:
Carfilzomib (Kyprolis)
Carfilzomib is a second-generation tetrapeptide epoxyketone proteasome inhibitor for multiple myeloma. FDA-approved 2012 (monotherapy), expanded 2015-2016 (combinations). ASPIRE Phase 3 (n=792): PFS 26.3 vs 17.6 months with KRd vs Rd (HR 0.69, P<0.001). OS improved to 48.3 vs 40.4 months. ENDEAVOR: PFS 18.7 vs 9.4 months vs bortezomib (HR 0.53). SAFETY: Significant cardiac toxicity - heart failure (4-16%), hypertension (9-27%), thromboembolism (24%). Requires cardiac monitoring.
- Best evidence:
Lixisenatide (Adlyxin)
Lixisenatide is a once-daily GLP-1 receptor agonist for type 2 diabetes. FDA-approved 2016 following ELIXA cardiovascular safety trial. GetGoal Phase 3 program (n>5,000): Significant HbA1c reductions across 13 trials. ELIXA (n=6,068): Confirmed CV safety (HR 1.02 for MACE, noninferiority P<0.001). Pronounced postprandial glucose effect - 75% reduction in glucose excursion (GetGoal-Mono). Effective add-on to basal insulin. Common side effects: nausea, vomiting (dose-dependent, resolve in 3-6 weeks).
- Best evidence:
Octreotide (Sandostatin)
Octreotide is a synthetic octapeptide analog of somatostatin with prolonged action. FDA-approved in 1988, it is available as immediate-release injection and long-acting repeatable (LAR) formulation. Primary indications include acromegaly, carcinoid syndrome, and neuroendocrine tumors. The PROMID trial demonstrated significant antiproliferative effects in midgut neuroendocrine tumors, and multiple trials confirm efficacy for GH/IGF-1 suppression in acromegaly.
- Best evidence:
Plitidepsin (Aplidin)
Plitidepsin is a cyclic depsipeptide originally from Mediterranean marine tunicate Aplidium albicans, now synthesized. FDA/EMA orphan drug status for multiple myeloma. Approved in Australia (2018) for relapsed/refractory MM. Phase III trials ongoing for breast and lung cancers. Targets eEF1A2 protein overexpressed in cancer. Phase I/II: Demonstrated antitumor activity with tolerable safety. Enhanced efficacy with dexamethasone combination. Safe cardiac profile (1.9% drug-related cardiac AEs in 578 patients). Also studied for COVID-19.
- Best evidence:
Retatrutide (GLP-3 Triple Agonist)
Retatrutide (also known as GLP-3 or LY3437943) is an investigational triple hormone receptor agonist (GIP/GLP-1/glucagon) by Eli Lilly for obesity, T2DM, and MASLD. Phase 2: 24.2% weight loss at 48 weeks (NEJM 2023). Phase 3 TRIUMPH-4: 28.7% weight loss at 68 weeks. T2DM Phase 2: HbA1c reduction of 2.2%, 82% achieved HbA1c ≤6.5%. MASLD: 86% liver fat reduction at 48 weeks, 93% achieved <5% liver fat. SAFETY: GI side effects common - nausea (43%), diarrhea (33%), vomiting (21%). Dysesthesia reported in ~20% at higher doses. Seven Phase 3 readouts expected 2026.
- Best evidence:
Teriparatide (Forteo)
Teriparatide (brand name Forteo) is a recombinant form of the first 34 amino acids of human parathyroid hormone (PTH 1-34). It was FDA-approved in November 2002 as the first anabolic agent for osteoporosis treatment. Unlike antiresorptive drugs, teriparatide stimulates new bone formation. It is indicated for postmenopausal women and men with osteoporosis at high fracture risk, and for glucocorticoid-induced osteoporosis.
- Best evidence:
Thymosin Alpha-1
Thymosin Alpha-1 (Tα1) is a 28-amino acid immunomodulatory peptide originally isolated from thymic tissue. It enhances T-cell function, modulates innate and adaptive immune responses, and has been approved in over 35 countries for treatment of chronic hepatitis B and as an immune adjuvant. Clinical research supports its use in viral hepatitis, sepsis, and as adjunctive cancer therapy, with a favorable safety profile across numerous trials.
- Best evidence:
Angiotensin II
Angiotensin II is the endogenous vasoconstrictor peptide now available as a synthetic IV formulation (Giapreza). FDA-approved 2017 for vasodilatory/septic shock refractory to standard vasopressors. A-GRADE evidence from ATHOS-3 (n=321): 70% achieved MAP response vs 23% placebo (P<0.001). Post-hoc analysis: AKI patients on RRT showed 53% vs 30% survival at day 28. SAFETY CONCERN: Increased thromboembolism (13% vs 5%), requires DVT prophylaxis. No mortality benefit in main analysis - approved for hemodynamic response, not survival. PRESCRIPTION ONLY - ICU setting.
- Best evidence:
Daptomycin (Cubicin)
Daptomycin is a cyclic lipopeptide antibiotic for serious gram-positive infections. FDA-approved 2003 (cSSSI) and 2006 (S. aureus bacteremia/endocarditis). First-in-class lipopeptide with rapid bactericidal activity against MRSA/MSSA. Phase 3: cSSSI success 83.4% vs 84.2% comparators (n=902). Bacteremia/endocarditis: 44.2% vs 41.7% standard therapy (noninferior, P met). Significantly less nephrotoxicity than vancomycin+gentamicin (11.0% vs 26.3%, P=0.004). SAFETY CONCERN: CPK elevation/myopathy (2.8%), monitor weekly; eosinophilic pneumonia (rare but serious); avoid with statins.
- Best evidence:
DSIP (Delta Sleep-Inducing Peptide)
DSIP (Delta Sleep-Inducing Peptide) is a naturally occurring nonapeptide (Trp-Ala-Gly-Gly-Asp-Ala-Ser-Gly-Glu) first isolated from rabbit brain in 1977. It has been studied for sleep regulation, stress protection, pain modulation, and substance withdrawal. While demonstrating promise in various clinical applications including opioid and alcohol withdrawal, its mechanisms remain incompletely understood, and human clinical data shows mixed results for sleep induction.
- Best evidence:
GHRP-6
GHRP-6 (Growth Hormone-Releasing Peptide-6) is a synthetic hexapeptide (His-DTrp-Ala-Trp-DPhe-Lys-NH2) that potently stimulates growth hormone secretion. Developed in the 1980s by Cyril Bowers, it was the first synthetic peptide shown to specifically elicit dose-dependent GH release both in vitro and in vivo. GHRP-6 acts through the ghrelin receptor (GHS-R1a) and CD36 receptor, exhibiting not only GH-releasing properties but also significant cytoprotective, cardioprotective, and wound healing effects. It increases IGF-1 levels, stimulates appetite, and has demonstrated tissue-protective properties in multiple preclinical and clinical studies. While primarily researched for GH deficiency, emerging evidence supports its potential in wound healing, scar prevention, and cardioprotection.
- Best evidence:
KPV
KPV (Lys-Pro-Val) is a C-terminal tripeptide fragment of alpha-melanocyte-stimulating hormone (α-MSH) that exhibits potent anti-inflammatory and immunomodulatory properties. Preclinical studies have demonstrated significant efficacy in reducing intestinal inflammation in multiple models of inflammatory bowel disease, including DSS- and TNBS-induced colitis. Unlike full-length α-MSH, KPV's anti-inflammatory action is not primarily melanocortin receptor-mediated but is instead transported into cells via the PepT1 transporter. No human clinical trials have been conducted to date, limiting the evidence to animal and in vitro studies.
- Best evidence:
Nesiritide (Natrecor)
Nesiritide is a recombinant form of human B-type natriuretic peptide (BNP). FDA-approved in 2001 for acute decompensated heart failure (ADHF). Clinical trials in >2000 patients demonstrated rapid hemodynamic improvement and dyspnea relief. The VMAC trial showed superiority over nitroglycerin for dyspnea improvement. Administered as IV bolus followed by continuous infusion.
- Best evidence:
Romidepsin (Istodax)
Romidepsin is a bicyclic depsipeptide histone deacetylase (HDAC) inhibitor derived from Chromobacterium violaceum. FDA-approved November 2009 for relapsed/refractory CTCL and June 2011 for PTCL. Phase 2 CTCL trials: ORR 34-35% with 6% CR. Phase 2 PTCL: ORR 25-38% with durable responses (median DOR 28 months). Acts as prodrug with intracellular disulfide reduction releasing zinc-binding thiol to inhibit HDACs.
- Best evidence:
Secretin (ChiRhoStim)
Secretin is a 27-amino acid peptide hormone. FDA-approved in 2004 for diagnostic use. Stimulates pancreatic bicarbonate secretion for exocrine function testing. Secretin stimulation test has 92-96% sensitivity for gastrinoma/ZES diagnosis. Used in secretin-enhanced MRCP with 89% sensitivity for chronic pancreatitis detection. Also facilitates ERCP cannulation.
- Best evidence:
Semax
Semax is a synthetic heptapeptide derived from ACTH (adrenocorticotropic hormone). It has been approved as a prescription medication in Russia and Ukraine for treating stroke, cognitive disorders, and peptic ulcers. Research suggests it may enhance BDNF expression and exhibit nootropic and neuroprotective effects. Most clinical research has been conducted in Russia, with limited Western clinical trials.
- Best evidence:
Degarelix (Firmagon)
Degarelix is a third-generation GnRH antagonist peptide. FDA-approved for advanced prostate cancer. Phase III trial (CS21) showed 97.2% achieved testosterone ≤0.5 ng/mL. Achieves castration in 96% of patients by day 3 (vs 0% with leuprolide). No testosterone flare or need for antiandrogen flare protection. Median PSA significantly lower at days 14 and 28 vs leuprolide (p<0.001).
- Best evidence:
Goserelin (Zoladex)
Goserelin is a synthetic decapeptide GnRH agonist. FDA-approved in 1989, it is used for prostate cancer, breast cancer, and endometriosis. Clinical trials show 82% objective response in metastatic prostate cancer and 10-year survival rates of 42-87% depending on setting. Available as 3.6mg (monthly) and 10.8mg (3-month) subcutaneous implants.
- Best evidence:
Leuprolide (Lupron)
Leuprolide is a synthetic nonapeptide GnRH receptor agonist. FDA-approved since 1985, it is used for prostate cancer, endometriosis, uterine fibroids, and central precocious puberty. Clinical trials show comparable efficacy to orchiectomy for prostate cancer and superior to placebo for endometriosis. Available as daily injection and depot formulations (1, 3, 4, and 6-month).
- Best evidence:
Nafarelin (Synarel)
Nafarelin is a synthetic GnRH agonist decapeptide with D-Nal(2) substitution at position 6. FDA-approved for endometriosis and central precocious puberty. Administered via nasal spray. Double-blind trials show >80% reduction in endometriosis extent, symptoms reduced from 40% severe to 5-10%. 39% pregnancy rate post-treatment. Also approved for CPP in children ages 8/9 and under.
- Best evidence:
Romosozumab (Evenity)
Romosozumab (Evenity) is a humanized monoclonal antibody that inhibits sclerostin, a key negative regulator of bone formation. FDA-approved in April 2019 for postmenopausal women with osteoporosis at high fracture risk. It has a unique dual mechanism: stimulating bone formation while reducing bone resorption. The FRAME and ARCH trials demonstrated significant reductions in vertebral and clinical fractures.
- Best evidence:
Setmelanotide (Imcivree)
Setmelanotide (brand name Imcivree) is an 8-amino-acid cyclic peptide that acts as a selective melanocortin-4 receptor (MC4R) agonist. It was FDA-approved in November 2020 as the first therapy for chronic weight management in patients with obesity due to POMC, PCSK1, or LEPR deficiency, and in 2022 for Bardet-Biedl syndrome (BBS). It works by restoring MC4R pathway signaling to reduce hyperphagia and promote weight loss.
- Best evidence:
Terlipressin (Terlivaz)
Terlipressin is a synthetic vasopressin analog with preferential V1 receptor activity used for hepatorenal syndrome and variceal bleeding. FDA-approved September 2022 as the first and only drug for HRS-AKI in the US. CONFIRM trial (n=300): HRS reversal 32% vs 17% placebo (P=0.012). Also highly effective for acute variceal bleeding - Cochrane meta-analysis showed 34% relative reduction in mortality vs placebo. SAFETY CONCERN: Black box warning for respiratory failure risk - avoid if oxygen saturation <90%.
- Best evidence:
Caspofungin (Cancidas)
Caspofungin is a cyclic lipopeptide echinocandin - the first in its class FDA-approved (2001). Indicated for invasive candidiasis, candidemia, esophageal candidiasis, and invasive aspergillosis. Phase 2/3 trials: 85% response in esophageal candidiasis vs 67% amphotericin B. Active against fluconazole-resistant Candida (MIC90 0.5-1 ug/ml for 157 resistant isolates). Safe profile with only 2% discontinuation in RCTs. Standard comparator for newer echinocandins.
- Best evidence:
Cetrorelix (Cetrotide)
Cetrorelix is a synthetic decapeptide GnRH antagonist. FDA-approved in 1999 for IVF/ICSI to prevent premature LH surge. Available as 0.25mg (multiple dose) and 3mg (single dose). Phase 3 trials show ~100% prevention of premature LH surge. Clinical pregnancy rate 26.3%. Nearly 100% prevention of ovarian hyperstimulation syndrome. Currently gold standard for IVF protocols.
- Best evidence:
GHK-Cu
GHK-Cu (Copper peptide GHK-Cu) is a naturally occurring tripeptide containing glycine, histidine, lysine, and a copper ion. It was first identified in human plasma and has been extensively studied for wound healing, tissue regeneration, and anti-aging applications. Research suggests it may stimulate collagen synthesis, promote wound healing, and exhibit anti-inflammatory properties.
- Best evidence:
Glucagon (GlucaGen/Gvoke/Baqsimi)
Glucagon is a 29-amino acid peptide hormone. FDA-approved in 1998 for severe hypoglycemia emergency treatment. Phase 3 trials show 99-100% treatment success. Median time to glucose recovery 10-13 minutes. Available as injectable kit, autoinjector (Gvoke), and nasal powder (Baqsimi). Approved for ages ≥2 years (injectable) and ≥4 years (nasal).
- Best evidence:
Gonadorelin (Factrel)
Gonadorelin is a synthetic decapeptide identical to endogenous GnRH. FDA-approved as diagnostic agent for evaluating pituitary gonadotrope function. Also used therapeutically for hypogonadotropic hypogonadism via pulsatile pump. In males with HH, pulsatile GnRH achieves spermatogenesis in 85% (58/68 patients). Earlier spermatogenesis vs gonadotropin therapy (6 vs 14 months, p=0.01).
- Best evidence:
Humanin
Humanin (HN) is a 24-amino-acid mitochondrial-derived peptide encoded within the 16S rRNA gene of mitochondrial DNA. Discovered in 2001 from preserved brain tissue of Alzheimer's patients, it demonstrates potent neuroprotective, cytoprotective, and metabolic regulatory effects. Humanin levels decline with age but remain stable in long-lived species and are elevated in offspring of centenarians.
- Best evidence:
Lutetium-177 Dotatate (Lutathera)
Lutetium-177 dotatate is a radiolabeled somatostatin analog for peptide receptor radionuclide therapy (PRRT). FDA-approved 2018 for SSTR-positive GEP-NETs. NETTER-1 Phase 3: 65.2% PFS at 20 months vs 10.8% control. 79% reduction in disease progression/death risk. Median OS 48 months vs 36.3 months. First radiopharmaceutical for NETs.
- Best evidence:
Micafungin (Mycamine)
Micafungin is a cyclic lipopeptide echinocandin antifungal. FDA-approved 2005 for esophageal candidiasis, invasive candidiasis/candidemia, and uniquely for Candida prophylaxis in HSCT recipients. Clinical trials (n=3028 pooled): Favorable safety profile. Superior to fluconazole for HSCT prophylaxis. Fungicidal against Candida including fluconazole-resistant strains. Second echinocandin approved in US after caspofungin.
- Best evidence:
Mifamurtide (Mepact)
Mifamurtide is a synthetic muramyl tripeptide immunomodulator for osteosarcoma. EMA-approved March 2009 (FDA denied 2007). Phase 3 INT-0133 (n=662): 6-year overall survival 78% vs 70% with chemotherapy alone. 28% relative reduction in death risk (HR 0.72, P=0.031). Indicated for high-grade, non-metastatic, resectable osteosarcoma ages 2-30 combined with post-operative chemotherapy. First immunotherapy approved for osteosarcoma. LIMITATIONS: Not FDA-approved; EMA approval based on single trial with statistical controversies.
- Best evidence:
Pasireotide (Signifor)
Pasireotide is a novel multi-receptor-targeted somatostatin analog with high affinity for somatostatin receptor subtypes 1, 2, 3, and 5. FDA-approved in 2012, it is the first pituitary-directed medication for Cushing's disease. Available as twice-daily subcutaneous injection (Signifor) and long-acting monthly injection (Signifor LAR) for acromegaly. The Phase III PASPORT trial demonstrated significant urinary free cortisol normalization in Cushing's disease patients.
- Best evidence:
Pegcetacoplan (Empaveli)
Pegcetacoplan is a PEGylated 15-amino acid cyclic peptide that inhibits complement C3. FDA-approved May 2021 for adults with paroxysmal nocturnal hemoglobinuria (PNH). First C3-targeted PNH therapy. Phase III PEGASUS trial: Superior to eculizumab for hemoglobin improvement (p<0.001); 85% vs 15% transfusion-free at 16 weeks. Inhibits both intravascular and extravascular hemolysis. Derived from compstatin family of C3 inhibitors. SC infusion 1080 mg twice weekly.
- Best evidence:
Pegvisomant (Somavert)
Pegvisomant is a genetically modified growth hormone analog that functions as a GH receptor antagonist. FDA-approved in 2003, it is the first and only GH receptor antagonist for acromegaly. It is indicated for patients with inadequate response to surgery, radiation, or somatostatin analogs. Phase III trials showed up to 97% of patients achieving IGF-1 normalization with long-term treatment, making it the most effective medical therapy for biochemical control.
- Best evidence:
Polymyxin B
Polymyxin B is a cyclic lipopeptide antibiotic discovered in the 1940s from Paenibacillus polymyxa. Re-established as last-resort therapy for MDR/XDR gram-negative infections including carbapenem-resistant Enterobacteriaceae (CRE), MDR Pseudomonas aeruginosa, and MDR Acinetobacter baumannii. Multicenter studies (n=312): 70.5% 14-day survival for CRGNB infections. Less nephrotoxic than colistin. Non-renal elimination allows use without dose adjustment for renal impairment. SAFETY CONCERN: Dose-limiting nephrotoxicity (35-60% AKI incidence); narrow therapeutic window.
- Best evidence:
Pramlintide (Symlin)
Pramlintide is a synthetic analog of human amylin, a peptide hormone co-secreted with insulin from pancreatic beta cells. FDA-approved in 2005, it is used as adjunctive therapy to mealtime insulin in type 1 and type 2 diabetes. Clinical trials showed HbA1c reductions of 0.3-0.62% plus weight loss, with 48% achieving dual improvement in HbA1c and weight (vs 16% placebo).
- Best evidence:
Romiplostim (Nplate)
Romiplostim is a recombinant Fc-peptide fusion protein thrombopoietin receptor agonist. FDA-approved 2008 for chronic ITP, 2018 for pediatric ITP. Phase 3: 88% overall platelet response vs 14% placebo in non-splenectomized patients. Durable response in 52% pediatric patients vs 10% placebo. Weekly SC injection maintains platelets ≥50,000/μL.
- Best evidence:
Triptorelin (Trelstar)
Triptorelin is a synthetic decapeptide GnRH agonist. FDA-approved in 2000, available as 1-month (3.75mg), 3-month (11.25mg), and 6-month (22.5mg) formulations. Clinical trials show 97.5% achieve castrate testosterone by day 29 with 6-month formulation. 9-month survival significantly higher than leuprolide (97.0% vs 90.5%, p=0.033). Also approved for adjuvant breast cancer treatment.
- Best evidence:
Vasopressin (Vasostrict/Pitressin)
Vasopressin (arginine vasopressin, AVP) is a nonapeptide hormone. FDA-approved in 2014 for vasodilatory shock (septic shock, post-cardiotomy). VASST trial (NEJM 2008, n=778) showed mortality benefit in less severe septic shock (26.5% vs 35.7%, p=0.05). Allows reducing norepinephrine requirements. Dose: 0.01-0.1 units/minute. Surviving Sepsis Campaign recommends adding AVP when NE ≥0.25-0.50 µg/kg/min.
- Best evidence:
Abaloparatide
Abaloparatide (Tymlos) is an FDA-approved (2017) anabolic osteoporosis drug, NOT a supplement. It's a synthetic PTHrP analog that builds bone more effectively than older therapies. A-GRADE evidence for vertebral fracture reduction (86%), non-vertebral fractures (43%), and BMD increases. Requires daily subcutaneous injection. PRESCRIPTION ONLY - included here for informational purposes. Used in high-risk postmenopausal women and men. Has faster receptor kinetics than teriparatide with possibly better safety profile.
- Best evidence:
Atosiban
Atosiban is a nonapeptide oxytocin/vasopressin receptor antagonist for preterm labor. EMA-approved 2000, used in 68+ countries (NOT FDA-approved in US). A-GRADE evidence: Significantly more patients undelivered at 48h and 7 days vs placebo (P≤0.008) at ≥28 weeks gestation. Similar efficacy to beta-agonists but dramatically better tolerated (7.9% vs 70.8% adverse events vs ritodrine). Cochrane review: No superiority vs placebo or other tocolytics for NEONATAL outcomes - delays delivery but doesn't improve baby outcomes. CAUTION at <28 weeks (one trial noted increased fetal deaths). PRESCRIPTION ONLY.
- Best evidence:
Colistin (Polymyxin E)
Colistin is a cyclic lipopeptide antibiotic (polymyxin E) discovered 1947, FDA-approved 1959. Administered as inactive prodrug colistimethate sodium (CMS) for parenteral use. WHO Essential Medicine and critically important for human medicine. Last-resort therapy for MDR gram-negative infections. Meta-analysis of 5 RCTs (n=377): 36% nephrotoxicity incidence vs 15% comparators (NNH=5). Pediatric review (17 studies, n=312): Effective for BSI, pneumonia, meningitis in neonates. SAFETY CONCERN: Dose-dependent nephrotoxicity (40-45% incidence); neurotoxicity reported; 75% AKI recovery upon discontinuation.
- Best evidence:
Enfuvirtide (Fuzeon)
Enfuvirtide is a 36-amino acid synthetic peptide and the first FDA-approved HIV fusion inhibitor (2003). Derived from gp41, it blocks HIV entry into CD4 cells. Phase III trials showed superior viral suppression vs background regimen alone in treatment-experienced patients. Administered as 90mg subcutaneous injection twice daily.
- Best evidence:
Etelcalcetide (Parsabiv)
Etelcalcetide is a synthetic D-amino acid peptide calcimimetic approved for secondary hyperparathyroidism (SHPT) in hemodialysis patients. FDA-approved in 2017, it is the first IV calcimimetic. Phase III trials showed 74-75% of patients achieved >30% PTH reduction vs 8-10% placebo. Administered via IV injection at end of hemodialysis 3x/week.
- Best evidence:
Histrelin (Vantas/Supprelin)
Histrelin is a nonapeptide GnRH agonist. FDA-approved 1991 (daily), 2004 (implant for prostate cancer), 2007 (implant for CPP). Once-yearly subcutaneous implant releases 65 mcg/day. Phase III trials show 100% achieve testosterone ≤50 ng/dL within 4 weeks. Mean testosterone 13.1 ng/dL maintained through repeated cycles. PSA decreased 90% from baseline. Also approved for central precocious puberty.
- Best evidence:
LL-37
LL-37 (Leucine-Leucine-37) is the only human cathelicidin antimicrobial peptide, a 37-amino acid sequence cleaved from the C-terminus of the precursor protein hCAP-18. It is produced by neutrophils, epithelial cells, and other immune cells and plays crucial roles in innate immunity, wound healing, and antimicrobial defense. Clinical trials have demonstrated efficacy in treating hard-to-heal venous leg ulcers, while extensive preclinical research shows potent activity against antibiotic-resistant bacteria including MRSA biofilms.
- Best evidence:
Sincalide (Kinevac)
Sincalide is the synthetic C-terminal octapeptide of cholecystokinin (CCK-8). FDA-approved in 1976 for diagnostic imaging. Stimulates gallbladder contraction for ejection fraction measurement. GBEF <38% (60-min infusion) indicates dysfunction. 81% symptom relief post-cholecystectomy in dyskinesia patients. Also used for pancreatic function testing with secretin.
- Best evidence:
Vasoactive Intestinal Peptide (VIP/Aviptadil)
VIP is a 28-amino acid neuropeptide with potent immunomodulatory, anti-inflammatory, and vasodilatory properties. Aviptadil is the synthetic form (RLF-100). FDA orphan drug designation for pulmonary conditions. Preclinical: Highly effective in arthritis, IBD, and autoimmune models. Clinical trials for pulmonary hypertension showed hemodynamic improvements. COVID-19 ARDS trials showed mixed results - improved survival in small trial but negative in larger TESICO trial (n=461). Major limitation: short plasma half-life requiring specialized delivery.
- Best evidence:
Voclosporin (Lupkynis)
Voclosporin is a novel cyclic peptide calcineurin inhibitor for lupus nephritis. FDA-approved January 2021. First oral therapy approved for active lupus nephritis. Phase 3 AURORA 1 (n=357): Complete renal response 40.8% vs 22.5% placebo at 52 weeks (OR 2.7, P<0.001). UPCR ≤0.5 achieved by 45.3% vs 23.0%. AURORA 2 extension: Safety and efficacy maintained at 3 years. Cyclosporine analogue with consistent PK profile - no therapeutic drug monitoring required. SAFETY: Hypertension, nephrotoxicity (monitor eGFR), serious infections. Use with MMF and low-dose steroids.
- Best evidence:
Afamelanotide
Afamelanotide (Scenesse) is an FDA-approved (2019) prescription drug for erythropoietic protoporphyria (EPP), a rare genetic photosensitivity disorder. It's a synthetic α-MSH analog that stimulates eumelanin production WITHOUT UV exposure. A-GRADE evidence for increasing pain-free sun exposure and reducing phototoxic reactions. B-GRADE for vitiligo repigmentation (with NB-UVB). This is a PRESCRIPTION IMPLANT, not a tanning supplement. Related to but distinct from illegal 'Melanotan II'.
- Best evidence:
Bivalirudin
Bivalirudin is a synthetic 20-amino acid peptide derived from hirudin (leech anticoagulant) that directly inhibits thrombin. FDA-approved 2000 for PCI anticoagulation, including patients with HIT/HITTS. A-GRADE evidence: HORIZONS-AMI showed reduced major bleeding (4.9% vs 8.3% vs heparin+GPI). Over 25,000 patients studied across REPLACE-2, ACUITY, and HORIZONS-AMI trials. B-GRADE for mortality reduction (driven by bleeding reduction). SAFETY CONCERN: Increased acute stent thrombosis within 24h (1.3% vs 0.3% in HORIZONS-AMI) - mitigated by post-PCI infusion. ESC downgraded to Class IIb in 2018. PRESCRIPTION ONLY - catheterization lab setting.
- Best evidence:
CJC-1295
CJC-1295 is a synthetic analog of growth hormone-releasing hormone (GHRH) consisting of 29 amino acids with modifications to extend its biological half-life. Developed by ConjuChem Biotechnologies, it exists in two forms: CJC-1295 with DAC (Drug Affinity Complex) which binds to albumin for an extended half-life of 6-8 days, and Modified GRF (1-29) without DAC with a half-life of approximately 30 minutes. Clinical trials demonstrated sustained, dose-dependent increases in growth hormone and IGF-1 levels in healthy adults. Development was discontinued after Phase II trials, and it remains an investigational compound not approved by the FDA.
- Best evidence:
Gramicidin
Gramicidin is a cyclic peptide antibiotic from Bacillus brevis, first isolated 1941, FDA-approved 1955 as Neosporin component. Topical use only (lozenges, eye drops, ointments) due to hemolysis. Gramicidin D (mixture of A/B/C) used clinically. Ophthalmic study (n=91): Effective and safe for bacterial corneal ulceration. Combined with polymyxin B and neomycin. One of first membrane-active peptide antibiotics. SAFETY CONCERN: Highly hemolytic - systemic use contraindicated.
- Best evidence:
Ipamorelin
Ipamorelin is a synthetic pentapeptide (Aib-His-D-2-Nal-D-Phe-Lys-NH2) that acts as a selective growth hormone secretagogue. It stimulates growth hormone release from the pituitary gland by binding to the ghrelin/GHSR-1a receptor, with notable selectivity that does not significantly affect ACTH or cortisol levels, distinguishing it from other growth hormone releasing peptides.
- Best evidence:
Linaclotide (Linzess)
Linaclotide is a 14-amino acid peptide guanylate cyclase-C (GC-C) agonist. FDA-approved 2012 for chronic idiopathic constipation (CIC) and IBS-C. Phase 3: 33.7% FDA responder rate vs 13.9% placebo in IBS-C. Improves bowel frequency, stool consistency, and abdominal pain. Doses: 72-145 mcg (CIC), 290 mcg (IBS-C) once daily.
- Best evidence:
Oxytocin (Pitocin)
Oxytocin is a nonapeptide hormone. FDA-approved for labor induction and postpartum hemorrhage prevention. Cochrane review (61 studies, >12,000 women) confirms safety and efficacy. Reduces failure to deliver vaginally within 24h (8.4% vs 53.8% vs expectant management, RR 0.16). Standard of care for third stage of labor. Dosing: 0.5-20 mU/min IV for induction, 10 IU IM for PPH prevention.
- Best evidence:
Plecanatide (Trulance)
Plecanatide is a 16-amino acid synthetic uroguanylin analog and guanylate cyclase-C agonist. FDA-approved 2017 for CIC, 2018 for IBS-C. Phase 3: 21% durable CSBM responders vs 10.2% placebo in CIC (P<0.001). 30% overall responders vs 17.8% placebo in IBS-C. Lower diarrhea rate (5%) than linaclotide. Dose: 3 mg once daily.
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PT-141 (Bremelanotide)
PT-141 (Bremelanotide, brand name Vyleesi) is a synthetic cyclic heptapeptide melanocortin receptor agonist derived from the tanning peptide Melanotan II. It was FDA-approved in June 2019 for treating hypoactive sexual desire disorder (HSDD) in premenopausal women. Unlike PDE5 inhibitors, PT-141 works through central nervous system pathways to enhance sexual desire rather than peripheral blood flow.
- Best evidence:
Teduglutide (Gattex)
Teduglutide is a synthetic analog of glucagon-like peptide-2 (GLP-2) approved for the treatment of short bowel syndrome (SBS) in patients dependent on parenteral support. FDA-approved in 2012, it is the first and only GLP-2 analog available. Phase III trials demonstrated 65% response rate with 10% achieving parenteral nutrition independence. The glycine substitution at position 2 provides resistance to DPP-4 degradation.
- Best evidence:
Ziconotide (Prialt)
Ziconotide is a synthetic 25-amino acid peptide derived from omega-conotoxin MVIIA found in Conus magus marine snail venom. FDA-approved in 2004 for severe chronic pain refractory to other treatments. Administered intrathecally via implanted pump. RCTs showed significant pain reduction (14.7% vs 7.2% placebo). Unique non-opioid mechanism with no tolerance development.
- Best evidence:
Bacitracin
Bacitracin is a cyclic polypeptide antibiotic from Bacillus licheniformis, FDA-approved 1948. Primarily TOPICAL use for gram-positive skin infections. Combination products (triple antibiotic with neomycin/polymyxin B) are OTC first-aid kit staples. B-GRADE evidence: Cardiac surgery review (n=1495) showed zero deep sternal wound infections with prophylactic use. SAFETY: Systemic use ABANDONED due to severe nephrotoxicity. Topical is safe but allergic contact dermatitis occurs in 7-9% with prolonged exposure. One of the oldest antibiotics still in widespread use.
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Cosyntropin (Cortrosyn/Synacthen)
Cosyntropin is a synthetic ACTH(1-24) peptide. FDA-approved for diagnosis of adrenocortical insufficiency. The 250mcg ACTH stimulation test is the gold standard diagnostic test. Meta-analysis shows 97% sensitivity at 95% specificity for primary adrenal insufficiency. Less immunogenic than full ACTH due to absence of amino acids 25-39. Half-life 15 minutes with peak cortisol at 30-60 minutes.
- Best evidence:
Dasiglucagon (Zegalogue)
Dasiglucagon is a 29-amino acid glucagon analog with 7 amino acid substitutions for improved aqueous stability. FDA-approved March 2021 for severe hypoglycemia in patients 6+ with diabetes. First ready-to-use aqueous glucagon formulation (no reconstitution needed). Phase 3 trial (n=170): Median time to recovery 10 min vs 40 min placebo (P<0.001). 99% achieved plasma glucose recovery within 15 min. Also being studied for congenital hyperinsulinism.
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Difelikefalin (Korsuva)
Difelikefalin is a peripherally-restricted selective kappa opioid receptor (KOR) agonist peptide. FDA-approved August 2021 as first treatment for moderate-to-severe CKD-associated pruritus in hemodialysis patients. KALM trials: 51% achieved ≥3-point itch reduction vs 28% placebo. No abuse potential. Administered IV 0.5 mcg/kg post-dialysis.
- Best evidence:
Eptifibatide (Integrilin)
Eptifibatide is a synthetic cyclic heptapeptide GP IIb/IIIa inhibitor that blocks platelet aggregation. FDA-approved 1998 for ACS and PCI. PURSUIT Phase 3 (n=10,948): 1.5% absolute reduction in death/MI/refractory ischemia at 30 days (14.2% vs 15.7%, P=0.04). ESPRIT: 37% relative reduction in death/MI/TVR at 6 months with coronary stenting. High-risk medication per ISMP. SAFETY: Increased bleeding risk (major bleeding 1.3% vs 0.4% in ESPRIT), thrombocytopenia (0.2%).
- Best evidence:
Ganirelix (Antagon/Orgalutran)
Ganirelix is a synthetic decapeptide GnRH antagonist. First FDA-approved GnRH antagonist in US (1999). Phase III trials show effective LH surge prevention with 0.25mg daily dose. Comparable pregnancy rates to GnRH agonist protocols (31.0% vs 33.9%). 2.4% OHSS rate vs 5.9% with agonists. Shorter treatment duration (5 vs 26 days) and fewer injections.
- Best evidence:
Icatibant (Firazyr)
Icatibant is a synthetic decapeptide and selective bradykinin B2 receptor antagonist. FDA-approved in 2011 for acute hereditary angioedema (HAE) attacks. Phase III FAST trials showed median symptom relief in 2 hours vs 12-19.8 hours placebo/comparator. Administered as 30mg subcutaneous injection, self-injectable by patients.
- Best evidence:
Kisspeptin-54
Kisspeptin-54 (KP-54) is the major circulating isoform of kisspeptin in humans, a 54-amino-acid neuropeptide encoded by the KISS1 gene. It is a critical regulator of reproductive function, acting as the primary stimulator of gonadotropin-releasing hormone (GnRH) secretion from the hypothalamus. Kisspeptin is essential for puberty onset and fertility, and clinical trials have shown promise for its use in IVF treatment, particularly for preventing ovarian hyperstimulation syndrome.
- Best evidence:
Peginesatide (Omontys)
Peginesatide is a synthetic PEGylated dimeric peptide erythropoiesis-stimulating agent for renal anemia. FDA-approved March 2012. WITHDRAWN February 2013 due to fatal anaphylaxis (0.02% rate). EMERALD trials (n=1608): Once-monthly peginesatide noninferior to epoetin for hemoglobin maintenance in dialysis patients. PEARL trials: Cardiovascular events and mortality INCREASED in non-dialysis CKD patients vs darbepoetin. Unique: No structural homology to erythropoietin - effective even with anti-EPO antibodies. SAFETY CONCERN: Fatal anaphylactic reactions led to worldwide market withdrawal. For educational/historical reference only.
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Selank
Selank is a synthetic heptapeptide derived from tuftsin, an immunomodulatory peptide. It has been approved in Russia as a treatment for anxiety and neurasthenic conditions. Research indicates it may have anxiolytic, nootropic, and immunomodulatory properties without sedative effects. Like Semax, most clinical research has been conducted in Russia.
- Best evidence:
Telavancin (Vibativ)
Telavancin is a semi-synthetic lipoglycopeptide antibiotic with dual mechanism of action. FDA-approved September 2009 (cSSSI) and June 2013 (HABP/VABP). ATLAS trials (n=1794): Clinical cure 88.3% vs 87.1% vancomycin (noninferior) for cSSSI. ATTAIN trials: Approved for hospital-acquired pneumonia when alternatives not suitable. Coverage: MRSA, MSSA, Streptococci, E. faecalis. SAFETY CONCERN: Higher mortality in patients with pre-existing renal impairment; use when alternatives not suitable.
- Best evidence:
Anidulafungin
Anidulafungin is a cyclic lipopeptide echinocandin antifungal for invasive candidiasis and candidemia. FDA-approved 2006. A-GRADE evidence: Phase 3 trial showed 76% success vs 60% fluconazole (P=0.01). Potent activity against Candida albicans, C. glabrata, C. tropicalis, C. krusei (MIC90 0.06-0.12 μg/ml). Crucially, 99% of fluconazole-resistant isolates remain susceptible. No hepatic or renal dose adjustment needed due to non-CYP450 metabolism - advantageous in critically ill patients. PRESCRIPTION ONLY - IV administration.
- Best evidence:
Dalbavancin (Dalvance)
Dalbavancin is a second-generation lipoglycopeptide antibiotic for acute bacterial skin infections. FDA-approved May 2014. Once-weekly or single-dose therapy: 1500 mg single dose OR 1000 mg + 500 mg at 1 week. DISCOVER-1 & DISCOVER-2 Phase 3: Noninferior to vancomycin/linezolid 14-day course (79.7% vs 79.8%). Half-life >1 week enables weekly dosing. Coverage: MRSA (MIC <0.125 µg/mL), MSSA, Streptococci. Pediatric approval 2021 (birth and older). First single-dose complete ABSSSI treatment.
- Best evidence:
Ecallantide (Kalbitor)
Ecallantide is a 60-amino acid recombinant polypeptide kallikrein inhibitor for hereditary angioedema. FDA-approved December 2009. First subcutaneous HAE treatment approved. EDEMA3 & EDEMA4 Phase 3 (n=168): Significantly greater symptom improvement at 4h vs placebo (P<0.01). Treatment outcome score 53.4 vs 8.1 placebo (P=0.003). Effect apparent within 2h and maintained through 24h. SAFETY CONCERN: Anaphylaxis risk 3.9% - must be administered by healthcare professional with anaphylaxis treatment available.
- Best evidence:
Protirelin (TRH)
Protirelin is a synthetic tripeptide identical to hypothalamic thyrotropin-releasing hormone (TRH). FDA-approved for diagnostic assessment of thyroid function. Differentiates central from primary hypothyroidism. 90% of TSH-secreting adenomas show blunted TRH response vs normal response in thyroid hormone resistance. Peak TSH response at 20-30 minutes. Half-life ~5 minutes.
- Best evidence:
Rezafungin (Rezzayo)
Rezafungin is a second-generation cyclic lipopeptide echinocandin antifungal. FDA-approved March 2023 for candidemia and invasive candidiasis - first new echinocandin in over a decade. ReSTORE Phase 3 trial (n=199): Noninferior to caspofungin for day-14 global cure and 30-day mortality. Weekly dosing (vs daily for other echinocandins) enables outpatient treatment. Inhibits (1,3)-beta-D-glucan synthase to disrupt fungal cell wall.
- Best evidence:
Tyrothricin
Tyrothricin is a cyclic peptide antibiotic complex from Bacillus brevis - the first peptide antibiotic used in humans (1939). Contains 60-80% tyrocidine (cyclic decapeptides) and 20-40% gramicidin (linear peptides). Over 60 years of clinical use with no significant resistance reported. RCT (DoriPha, n=321): Tyrothricin lozenges showed 64% improved complete remission vs placebo at 72 hours for acute pharyngitis. Used topically for sore throat, skin infections. SAFETY CONCERN: Systemic toxicity limits to topical/local use only.
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Vosoritide (Voxzogo)
Vosoritide is a 39-amino acid C-type natriuretic peptide (CNP) analog for achondroplasia. FDA-approved November 2021 - first pharmacological treatment for achondroplasia. Phase 3 trial (n=121): Significant increase in annualized growth velocity vs placebo at 52 weeks. Daily SC injection promotes endochondral bone growth by antagonizing FGFR3 downstream signaling. Approved for children 5+ years with open epiphyses.
- Best evidence:
Macimorelin (Macrilen)
Macimorelin is an oral ghrelin receptor agonist peptidomimetic. FDA-approved in 2017 as first oral diagnostic for adult GH deficiency. Single dose 0.5 mg/kg. Phase 3: 82-87% sensitivity, 92-96% specificity at 2.7-5.1 ng/mL GH cutoff. Comparable accuracy to insulin tolerance test without hypoglycemia risk.
- Best evidence:
Motixafortide (Aphexda)
Motixafortide is a 14-amino acid cyclic peptide CXCR4 inhibitor for hematopoietic stem cell mobilization. FDA-approved September 2023 in combination with G-CSF for autologous transplant in multiple myeloma. First new stem cell mobilization agent in 15 years. GENESIS Phase 3 trial (n=122): 67.5% achieved collection goal of 6x10^6 CD34+ cells/kg in 2 apheresis sessions vs 9.5% placebo (P<0.0001). High-affinity (Ki 0.32 nM) with sustained mobilization effect (>48 hours).
- Best evidence:
Oritavancin (Orbactiv)
Oritavancin is a second-generation lipoglycopeptide antibiotic for acute bacterial skin infections. FDA-approved August 2014. Single-dose therapy: 1200 mg IV infusion provides complete treatment. SOLO-1 & SOLO-2 Phase 3 (n=1987): Noninferior to 7-10 day vancomycin course (81.2% vs 80.9% composite endpoint). Superior MRSA lesion reduction (93.1% vs 87.1%, P=0.032). Half-life 245 hours enables once-only dosing. Coverage: MRSA, MSSA, Streptococci, E. faecalis. Vancomycin derivative with enhanced lipophilic properties.
- Best evidence:
AOD-9604
AOD-9604 is a synthetic peptide fragment of human growth hormone (amino acids 176-191) developed for weight loss. DEVELOPMENT TERMINATED in 2007 after Phase 2b trials showed modest, inconsistent results. C-grade for small weight loss (~2kg over 12 weeks). Despite being abandoned by its developers, it's sold in gray market peptide circles. BANNED by WADA. Not FDA-approved for any use. Animal studies showed better results than human trials.
- Best evidence:
Dihexa (PNB-0408)
Dihexa (N-hexanoic-Tyr-Ile-(6) aminohexanoic amide) is an angiotensin IV-derived peptide that potentiates hepatocyte growth factor (HGF)/c-Met receptor signaling. Preclinical studies show remarkable cognitive enhancement at picomolar concentrations. In Alzheimer's disease mouse models, dihexa restored cognitive function and promoted synaptogenesis. Seven orders of magnitude more potent than BDNF in promoting dendritic spine formation. Currently investigational with no completed human clinical trials.
- Best evidence:
Pinealon
Pinealon is a synthetic tripeptide (Glu-Asp-Arg) developed by Russian researchers as part of the Khavinson peptide bioregulators. It is designed to target brain tissue and has been studied for potential neuroprotective and cognitive-enhancing effects. Research suggests it may help regulate circadian rhythms and support cognitive function in aging, though human clinical data is limited.
- Best evidence:
Corticorelin Ovine (Acthrel)
Corticorelin is a synthetic ovine corticotropin-releasing hormone (CRH) analog. FDA-approved for differentiating pituitary vs ectopic ACTH in Cushing's syndrome. 88% sensitivity, 100% specificity for Cushing's disease when using cortisol response criteria. Ovine CRH superior to human CRH. Positive response: >35% ACTH increase and >20% cortisol increase.