Papers and official documents

Wilding et al., STEP 1 · 2021

Once-weekly semaglutide in adults with overweight or obesity. N Engl J Med. 2021. PMID 33567185. DOI 10.1056/NEJMoa2032183. n=1,961; 68 weeks; mean weight change −14.9% vs −2.4%. N Engl J Med. PMID 33567185.

Lincoff et al., SELECT · 2023

Semaglutide and cardiovascular outcomes in obesity without diabetes. N Engl J Med. 2023. PMID 37952131. DOI 10.1056/NEJMoa2307563. n=17,604; primary MACE HR 0.80. N Engl J Med. PMID 37952131.

Perkovic et al., FLOW · 2024

Effects of semaglutide on chronic kidney disease in patients with type 2 diabetes. N Engl J Med. 2024. PMID 38785209. DOI 10.1056/NEJMoa2403347. n=3,533; once-weekly 1.0 mg; kidney composite HR 0.76. Labelled Ozempic CKD use, not an obesity score. N Engl J Med. PMID 38785209.

Sanyal et al., ESSENCE part 1 · 2025

Phase 3 trial of semaglutide in metabolic dysfunction-associated steatohepatitis. N Engl J Med. 2025. PMID 40305708. DOI 10.1056/NEJMoa2413258. NCT04822181. Week-72 histology in the first 800 patients. Supports Wegovy’s accelerated MASH indication; not an extra obesity score. N Engl J Med. PMID 40305708.

Ozempic (semaglutide) prescribing information

DailyMed setid adec4fd2-6858-4c99-91d4-531f5f2a2d79. Labelled to reduce the risk of sustained eGFR decline, end-stage kidney disease, and cardiovascular death in adults with type 2 diabetes and CKD, in addition to glycemic and CV uses. Pregnancy: use only if the potential benefit justifies the potential risk to the fetus (DailyMed 8.1).

Wegovy (semaglutide) prescribing information

DailyMed setid ee06186f-2aa3-4990-a760-757579d8f77b. Weight and CV labelled uses. Accelerated-approval indication for noncirrhotic MASH with moderate to advanced fibrosis. Pregnancy language differs for the MASH use.

FDA, Wegovy MASH accelerated approval

FDA notice, 15 August 2025. Accelerated approval of Wegovy injection for noncirrhotic MASH with moderate to advanced fibrosis. Other labelled use, not an obesity score.

SURMOUNT-OSA · 2024

Malhotra et al. Tirzepatide for the treatment of obstructive sleep apnea and obesity. Two phase 3 trials; primary endpoint change in AHI at 52 weeks. N Engl J Med. PMID 38912654.

FDA Approves First Medication for Obstructive Sleep Apnea

FDA press announcement: Zepbound approved for moderate to severe OSA in adults with obesity (20 December 2024).

Zepbound DailyMed SPL

Current Zepbound prescribing information: indications include OSA in adults with obesity; §4 MTC/MEN2 and hypersensitivity; §8.1 pregnancy.

SURMOUNT-1 · 2022

Jastreboff et al. Tirzepatide once weekly for the treatment of obesity. N Engl J Med. PMID 35658024.

SURPASS-2 · 2021

Frías et al. Tirzepatide versus semaglutide once weekly in type 2 diabetes. N Engl J Med. PMID 34170647.

Jastreboff et al., phase 2 · 2023

Triple-hormone-receptor agonist retatrutide for obesity — a phase 2 trial. N Engl J Med. 2023. PMID 37366315. DOI 10.1056/NEJMoa2301972. n=338; primary endpoint at 24 weeks; 48-week 12 mg arm −24.2% vs −2.1%. N Engl J Med. PMID 37366315.

Eli Lilly, TRIUMPH-2 and TRIUMPH-3 topline

Company announcement, 23 July 2026. Topline results, not a peer-reviewed paper. Cited only to identify that fact. A planned BLA in Q1 2027 is not an approval.

DCCT · 1993

Diabetes Control and Complications Trial Research Group. The effect of intensive treatment of diabetes. N Engl J Med. PMID 8366922.

ADA Standards of Care

Pharmacologic approaches to glycemic treatment — annual living standards.

Falutz et al. NEJM 2007 · 2007

Metabolic effects of a growth hormone-releasing factor in patients with HIV. Study 1 / NCT00123253 publication; VAT primary. N Engl J Med. PMID 18057338.

EGRIFTA WR DailyMed SPL

Tesamorelin for injection 11.6 mg/vial. Indication, limitations, §4 contraindications, WR vs SV not substitutable, Study 1/2 VAT data.

EGRIFTA SV DailyMed SPL

Tesamorelin for injection 2 mg/vial. Same labelled HIV-lipodystrophy VAT indication and aligned §4 / clinical-studies bridge.

Neer et al. · 2001

Effect of parathyroid hormone (1-34) on fractures and bone mineral density in postmenopausal osteoporosis. N Engl J Med. PMID 11346808.

Forteo label

FDA prescribing information.

RECONNECT · 2019

Kingsberg et al. Bremelanotide for the treatment of hypoactive sexual desire disorder. Obstet Gynecol. PMID 31599840.

Vyleesi label

FDA prescribing information.

ACOG

Clinical guidance on labor induction and oxytocin.

Intranasal reviews

Heterogeneous CNS trial literature; failed replications in several high-profile lines.

Preclinical tendon review · 2019

Gwyer, Wragg, Wilson. Gastric pentadecapeptide BPC 157 and its role in accelerating musculoskeletal soft tissue healing. Cell Tissue Res. 2019. Cell Tissue Res. PMID 30915550.

FDA Category 2 / withdrawn nominations

BPC-157 appears under bulk drug substances nominated but withdrawn, with immunogenicity, impurity, characterization, and limited safety-information notes.

PCAC July 23–24 2026 calendar

Agenda lists BPC-157 (free base) and BPC-157 acetate for ulcerative colitis toward the 503A Bulks List. Committee discussion is not a listing.

FDA PCAC slide deck

Nominations withdrawn; FDA evaluating at its discretion. FDA proposes BPC-157 (free base) and BPC-157 acetate NOT be included on the 503A Bulks List.

FDA BPC-157 PCAC briefing

BPC-157-related bulk drug substances briefing for the July 2026 PCAC; notes withdrawn nominations and FDA evaluation at its discretion for 503A Bulks List consideration.

Tβ4 wound biology · 2005

Goldstein, Hannappel, Kleinman. Thymosin β4: actin-sequestering protein moonlights in repair. Trends Mol Med. PMID 15967722.

WADA prohibited list

Thymosin beta-4 and fragments are prohibited in sport.

Teichman et al. · 2006

Prolonged stimulation of growth hormone and IGF-1 by CJC-1295 in healthy adults. J Clin Endocrinol Metab. PMID 16804041.

Tesamorelin contrast

The approved GHRH analog — see Egrifta label.

Raun et al. · 1998

Ipamorelin, the first selective growth hormone secretagogue. Eur J Endocrinol. PMID 9849822.

FDA Category 2

Ipamorelin acetate — immunogenicity, characterization, published IV serious adverse events including death.

Pickart & Margolina · 2018

GHK-Cu peptide in skin remodeling and anti-oxidative defense. Biomed Res Int. PMID 30498412.

Cosmetic ingredient

Copper tripeptide-1 in topical products — a cosmetic, not a NDA.

Gusev EI et al. · 2005

Semax in prevention of disease progress and development of exacerbations in patients with cerebrovascular insufficiency. Zh Nevrol Psikhiatr Im S S Korsakova. PMID 15792140.

PCAC July 2026 calendar

Meeting announcement. A committee discussion is not a 503A listing.

Seredenin et al.

Anxiolytic tuftsin analog series from the Institute of Pharmacology.

Evidence standard

No FDA-reviewed anxiety indication; no large Western RCTs.

Khavinson et al. · 2014

Peptide regulation of gene expression and telomerase activity (AEDG). Bull Exp Biol Med. PMID 25403401.

Evidence standard

Lacks contemporary, independently replicated outcome RCTs.

King & Tuthill · 2016

Clinical applications of thymosin alpha-1 — hepatitis B and immune-adjuvant programs. Expert Opin Biol Ther. PMID 27392153.

Zadaxin literature

Approved in various countries; not a US NDA for commercial use.

Lee et al. · 2015

The mitochondrial-derived peptide MOTS-c promotes metabolic homeostasis and reduces obesity and insulin resistance. Cell Metab. PMID 25738455.

Human observational follow-up

Circulating MOTS-c versus age and insulin sensitivity — not an interventional literature.

FDA accelerated approval notice, 19 September 2025 · 2025

FDA Grants Accelerated Approval to First Treatment for Barth Syndrome. Forzinity (elamipretide) injection. NDA 215244. Patients weighing at least 30 kg. Intermediate endpoint: knee extensor muscle strength. Confirmatory trial required.

Forzinity (elamipretide) prescribing information · 2025

DailyMed label, NDA 215244. Indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg. Accelerated approval based on knee extensor muscle strength. Setid 146bf34c-76f2-48db-ac07-fb29cce2cd75.

Reid Thompson et al., TAZPOWER · 2021

A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate elamipretide in Barth syndrome. Genet Med. 2021. Randomized period missed primary endpoints. PMID 33077895. DOI 10.1038/s41436-020-01006-8. Genet Med. PMID 33077895.

Karaa et al., MMPOWER-3 · 2023

Efficacy and safety of elamipretide in primary mitochondrial myopathy. Neurology. 2023. Missed primary endpoints (6MWT and fatigue). PMID 37268435. DOI 10.1212/WNL.0000000000207402. Neurology. PMID 37268435.

Szeto, cardiolipin-protective SS-31 review · 2014

First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics. Br J Pharmacol. 2014. PMID 24117165. DOI 10.1111/bph.12461. Br J Pharmacol. PMID 24117165.

Dermatology RCTs · 2014

Proksch et al. Oral collagen peptide supplementation and skin elasticity. Skin Pharmacol Physiol. PMID 23949208.

Joint-pain reviews

Mixed, modest effects across osteoarthritis trials — heterogeneity of source and dose is high.

Dorr et al. · 1996

Evaluation of melanotan-II, a superpotent cyclic melanotropic peptide, in a pilot phase-I study. Life Sci. PMID 8761332.

Dermatology case series

Nevus change and pigment events after unregulated tanning-peptide use.

Scenesse contrast

Approved afamelanotide for EPP is not MT-II.

Dhillo et al. · 2005

Kisspeptin-54 stimulates the hypothalamic-pituitary-gonadal axis in human males. J Clin Endocrinol Metab. PMID 16234307.

FDA Category 2

Kisspeptin-10 — immunogenicity and insufficient safety information for proposed compounding routes.

Obesity development

Metabolic Pharmaceuticals-era AOD-9604 trials did not achieve approval.

WADA

GH fragments listed among prohibited agents.

Cathelicidin dual role · 2006

Dürr, Sudheendra, Ramamoorthy. LL-37, the only human member of the cathelicidin family. Biochim Biophys Acta. PMID 16616139.

Rosacea literature · 2007

Yamasaki et al. Increased serine protease activity and cathelicidin in rosacea. Nat Med. PMID 17676051.

FDA approval notice — Yorvipath for hypoparathyroidism · 2024

FDA approves new drug for hypoparathyroidism, a rare disorder. Yorvipath (palopegteriparatide) for adults; not studied in acute post-surgical hypoparathyroidism. PaTHway 26-week RCT summary (69% vs 5% composite responders). Orphan designation and priority review. Dated with approval 9 Aug 2024.

Yorvipath (palopegteriparatide) DailyMed label · 2026

Prescribing information. PTH(1-34) analog for hypoparathyroidism in adults. NDA 216490. Setid a1fa23fd-c434-443e-9a17-5750578c8602. Limitations of use and PTH-class osteosarcoma warning on label.

Khan et al., PaTHway 26-week · 2023

Efficacy and Safety of Parathyroid Hormone Replacement With TransCon PTH in Hypoparathyroidism: 26-Week Results From the Phase 3 PaTHway Trial. J Bone Miner Res. 2023. PMID 36271471. DOI 10.1002/jbmr.4726. NCT04701203. J Bone Miner Res. PMID 36271471.

Clarke et al., PaTHway 52-week · 2025

Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial. J Clin Endocrinol Metab. 2025. PMID 39376010. DOI 10.1210/clinem/dgae693. J Clin Endocrinol Metab. PMID 39376010.

PaTHway ClinicalTrials.gov · 2021

NCT04701203. A Trial Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Daily in Adults With Hypoparathyroidism. Phase 3; completed.

FDA approval notice — Yuviwel for pediatric achondroplasia · 2026

FDA approves drug for pediatric patients with most common form of dwarfism. Yuviwel (navepegritide) for ages ≥2 with achondroplasia and open epiphyses. Accelerated approval on AGV (~1.5 cm vs placebo over 52 weeks). Confirmatory final adult height study required. Content current as of 03/04/2026.

Yuviwel (navepegritide) DailyMed label · 2026

Prescribing information. CNP analog; accelerated approval on AGV. NDA 219164. Setid e01c1197-338f-414c-b804-808e129ebc8c. Transient hypotension class warning. §4 contraindications: none.

Savarirayan et al., APPROACH · 2026

Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial. JAMA Pediatr. 2026. PMID 41247754. DOI 10.1001/jamapediatrics.2025.4771. NCT05598320. JAMA Pediatr. PMID 41247754.

APPROACH ClinicalTrials.gov · 2022

NCT05598320. A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia. Phase 2/3; completed.

FDA NDA 219164 approval package · 2026

Approval package PDF for Yuviwel (navepegritide), NDA 219164Orig1s000. Approval date February 27, 2026. Indication: increase linear growth in pediatric patients ≥2 with achondroplasia with open epiphyses.

FDA press announcement — Mimrylo (rusfertide) approval · 2026

FDA Approves First Drug of Its Kind for Polycythemia Vera. Immediate release 28 Aug 2026. VERIFY n=293 Phase 3 summary; first-in-class hepcidin mimetic.

Mimrylo (rusfertide) prescribing information (DailyMed) · 2026

SPL setid 101aff01-31ed-4841-a8b6-0a5a8503189b. Indication: erythrocytosis in adults with PV. Section 4 contraindications: none. Mechanism: hepcidin mimetic / ferroportin blockade. Clinical studies: VERIFY NCT05210790.

VERIFY — ClinicalTrials.gov NCT05210790

Phase 3 study of rusfertide (PTG-300) in polycythemia vera. Enrollment n=293 actual. Primary completion listed; peer-reviewed primary manuscript not identified on PubMed at draft time.

Kremyanskaya et al., REVIVE (Phase 2) · 2024

Rusfertide, a Hepcidin Mimetic, for Control of Erythrocytosis in Polycythemia Vera. N Engl J Med. 2024. PMID 38381675. DOI 10.1056/NEJMoa2308809. NCT04057040. Phase 2 randomized withdrawal; not VERIFY. N Engl J Med. PMID 38381675.

Miller et al., TRANSCEND · 2026

Setmelanotide for the treatment of acquired hypothalamic obesity. N Engl J Med. 2026;395(2):138-150. PMID 42418774. DOI 10.1056/NEJMoa2512275. NCT05774756. N Engl J Med. PMID 42418774.

Imcivree (setmelanotide) prescribing information · 2026

DailyMed label, NDA 213793. Labelled for acquired hypothalamic obesity (age 4+), Bardet-Biedl syndrome (age 2+), and POMC, PCSK1, or LEPR deficiency confirmed by genetic testing (age 2+). Not indicated for general obesity. Setid 70c3ccf7-4df0-4c75-ba07-fede9970c8d9.

Rhythm, 19 March 2026 approval note · 2026

FDA approval of an expanded Imcivree indication for acquired hypothalamic obesity. Company announcement, 19 March 2026.

Clément et al., POMC/LEPR phase 3 · 2020

Setmelanotide in severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre phase 3 trials. Lancet Diabetes Endocrinol. 2020. PMID 33137293. DOI 10.1016/S2213-8587(20)30364-8. Lancet Diabetes Endocrinol. PMID 33137293.

Haqq et al., BBS phase 3 · 2022

Setmelanotide in Bardet-Biedl syndrome and Alström syndrome: multicentre randomised phase 3 trial. Lancet Diabetes Endocrinol. 2022. PMID 36356613. DOI 10.1016/S2213-8587(22)00277-7. Lancet Diabetes Endocrinol. PMID 36356613.