Regulatory coverage on Peptide News Digest tracks how the FDA, MHRA, EMA, and state agencies handle peptides — what they let through, what they pull, what they redefine.
The compounding fight has dominated 2025 and 2026. GLP-1s came off the FDA shortage list in early 2025; the agency moved compounded semaglutide and tirzepatide toward Category 2 on the 503A bulks list; and a wave of state legislation tried to either preserve or shut off telehealth access. The PCAC has spent meetings on BPC-157, GHK-Cu, and other research peptides that have built consumer demand without clinical infrastructure behind them.
Stories here name the agency, the substance, and the action. Browse the latest below, or jump to specific tags like #fda, #compounding, #peptide-policy, or #503a.
The FDA approved Eli Lilly's Inluriyo (imlunestrant) in combination with Verzenio (abemaciclib) on Friday, September 18, 2026 for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer whose disease progressed after at least one line of endocrine therapy. The FDA also approved the Guardant360 CDx assay as the companion diagnostic for detecting ESR1 mutations. The approval rests on the EMBER-3 trial, which enrolled 874 adults; in the 159-patient ESR1-mutated subgroup, median progression-free survival was 11.1 months with the combination versus 5.5 months with imlunestrant alone (hazard ratio 0.53). Objective response rates were 35% and 15%, respectively.
Novartis announced on Friday, September 18, 2026 that the CHMP adopted a positive opinion for Cosentyx (secukinumab) to treat polymyalgia rheumatica (PMR) in adults who have had an inadequate response to steroids or who relapse during steroid taper. Novartis says Cosentyx would be the first IL-17A inhibitor licensed in Europe for PMR. The opinion is based on the global Phase III REPLENISH trial, run in 27 countries, in which all primary and secondary endpoints were met across both the 300 mg and 150 mg arms; Cosentyx doubled sustained remission rates and delivered steroid-sparing effects versus placebo, with no new safety signals. The data were published in the New England Journal of Medicine, and the European Commission is expected to decide within about two months.
Zealand Pharma announced on Friday, September 18, 2026 that the EMA's CHMP recommended approval of Zeydovio (glepaglutide) for short bowel syndrome. Zeydovio is a long-acting GLP-2 analog given twice weekly by subcutaneous injection from a ready-to-use, single-dose autoinjector. In the Phase 3 EASE-1 trial of 106 patients dependent on parenteral support, two thirds of patients on glepaglutide had at least a 20% reduction in weekly parenteral support volume at 24 weeks and one in seven weaned off it entirely; twice-weekly glepaglutide cut weekly parenteral support by 5.13 liters versus 2.85 liters on placebo. Zealand called the opinion the first major advance in short bowel syndrome treatment in Europe in more than a decade and expects a European Commission decision in about 67 days. In the U.S., the EASE-5 trial is ongoing, and glepaglutide has FDA orphan drug designation.
Merck announced on Friday, September 18, 2026 that the CHMP recommended approval of Keytruda (pembrolizumab) with Padcev (enfortumab vedotin) as neoadjuvant treatment, continued after radical cystectomy as adjuvant treatment, for adults with resectable muscle-invasive bladder cancer; the recommendation also covers subcutaneous Keytruda. In the Phase 3 KEYNOTE-B15 (EV-304) trial, the regimen reduced the risk of an event-free survival event by 47% and the risk of death by 35% versus gemcitabine and cisplatin chemotherapy plus surgery, and the pathologic complete response rate was 55.8% versus 32.5%. Merck said it would be the first and only PD-1 inhibitor plus antibody-drug conjugate regimen for muscle-invasive bladder cancer regardless of cisplatin eligibility in the EU. A European Commission decision is expected by the fourth quarter of 2026, and the FDA approved the combination for this use in July 2026. Merck's partners on the regimen are Astellas and Pfizer.
Xenon Pharmaceuticals announced on Thursday, September 17, 2026 that it submitted a New Drug Application to the FDA for azetukalner, a Kv7 potassium channel opener, in focal seizures, supported by the Phase 2b X-TOLE and Phase 3 X-TOLE2 studies. The company also paused enrollment of new patients in its major depressive disorder and bipolar depression studies after an analysis of neuropsychiatric adverse events; the pause was put in place with its Data Safety Monitoring Board, is expected to be temporary, and does not affect enrolled participants. Xenon said the events were consistent with azetukalner's known safety and tolerability profile. X-NOVA2, its depression study, had enrolled about 80% of its initial 450-patient target, and topline data are now expected in the first quarter of 2027.
Pfizer Canada and BioNTech announced on Thursday, September 17, 2026 that Health Canada authorized the XFG variant-adapted Comirnaty vaccine for active immunization against COVID-19 in people aged 6 months and older. The authorization was based on the cumulative clinical, non-clinical, and real-world data the companies had already submitted. The companies said the vaccine will be available through various distribution channels in the coming days and weeks, and Pfizer Canada said it has enough inventory to meet its contractual commitments.
Nuvation Bio announced on Thursday, September 17, 2026 that the FDA approved a supplemental New Drug Application updating the IBTROZI (taletrectinib) label for locally advanced or metastatic ROS1-positive non-small cell lung cancer. The label now reports a median duration of response of 49.7 months in TKI-naive patients in the Phase 2 TRUST-I study, based on a median follow-up of 51 months and an August 2025 data cutoff that added 14 months of data from TRUST-I and TRUST-II. Nuvation said the approval came four months ahead of the PDUFA date and brought no changes to the label's safety sections. IBTROZI, which Nuvation describes as a CNS-active next-generation ROS1 inhibitor, was first approved on June 11, 2025; Innovent Biologics markets it in China as DOVBLERON.
Ultragenyx Pharmaceutical announced on Thursday, September 17, 2026 that the FDA granted standard full approval to FAYUVI (rebisufligene etisparvovec-hopf), a single-dose intravenous AAV9 gene therapy, for the neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function. The company calls it the first FDA-approved treatment for Sanfilippo syndrome type A and its second gene therapy approval. Treated children (n=17) had a 23.5-point higher cognitive score than natural-history controls (n=27), with follow-up of up to nearly 8 years and reduced cerebrospinal fluid heparan sulfate. Ultragenyx received a priority review voucher, expects to ship within 30 to 60 days, and makes FAYUVI in Bedford, Massachusetts and at Andelyn Biosciences in Columbus, Ohio. BioSpace reported a $3.95 million wholesale acquisition cost, an initial FDA rejection in July 2025 over manufacturing issues, and a 13% share gain on the day.
NICE published final draft guidance on Thursday, September 17, 2026 recommending Enhertu (trastuzumab deruxtecan, from Daiichi Sankyo and AstraZeneca) for adults with HER2-low advanced or metastatic breast cancer whose disease has progressed after treatment, a group NICE estimated at about 1,000 people. NICE had previously rejected the drug for this use on cost-effectiveness grounds. It said the reversal followed an agreed commercial solution, the higher cost-effectiveness thresholds it began using in April 2026, and a new method for assessing quality of life published on August 27, 2026. The guidance is open to appeal until 5pm on October 7, 2026.
On July 28, 2026, the FDA published 17 revised draft product-specific guidances for generic versions of peptide drugs, including semaglutide (Ozempic, Wegovy), tirzepatide (Mounjaro, Zepbound), liraglutide, glucagon, dasiglucagon, calcitonin salmon, pegcetacoplan, teriparatide, and vosoritide. The recommendations cover how sponsors should submit recombinant, synthetic, or semi-synthetic peptides as ANDAs, innate immune response testing, impurity thresholds, higher order structure assessment, and biological activity assessment. The agency also withdrew its May 2021 guidance on ANDAs for highly purified synthetic peptides that reference drugs of rDNA origin, saying it no longer reflects current scientific thinking. The public comment window closes on September 28, 2026.
Scholar Rock Inc. (NASDAQ: SRRK) announced Monday September 14, 2026 the commercial launch of ISEMBYLD (apitegromab-mstn) following FDA approval on September 11, 2026 for the treatment of spinal muscular atrophy (SMA) in adults and children two years of age and older who are receiving a survival motor neuron 2 (SMN2)-targeted background therapy (Biogen's Spinraza / nusinersen, Novartis's Zolgensma / onasemnogene abeparvovec, or Roche's Evrysdi / risdiplam). ISEMBYLD is a fully human IgG4 monoclonal antibody that binds to promyostatin and latent myostatin (a peptide that limits muscle growth) and inhibits the activation of myostatin, blocking myostatin signaling to preserve and build muscle. The FDA action makes ISEMBYLD the first-and-only muscle-targeted SMA therapy — an addition to rather than a replacement for the SMN2-targeted background therapies that address the underlying survival motor neuron gene deficiency. Approval was based on Phase 3 SAPPHIRE trial data demonstrating motor function improvement (Hammersmith Functional Motor Scale Expanded score) at 12 months versus placebo in patients on stable SMN2-directed therapy. The FDA granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations. Scholar Rock reported net product sales of $0 (pre-launch) as of Q2 2026 with priced at approximately $310,000 per patient annually.
Cogent Biosciences (NASDAQ: COGT) announced Tuesday September 15, 2026 that the FDA accepted the New Drug Application (NDA) for bezuclastinib (a selective oral tyrosine kinase inhibitor targeting KIT D816V) in patients with Advanced Systemic Mastocytosis (AdvSM), an aggressive form of the rare hematologic disorder driven by activating mutations in the KIT receptor. FDA assigned a PDUFA target action date of June 29, 2027. The agency communicated that no advisory committee is planned and no potential review issues have been identified. Application support: the APEX Phase 2 registration trial primary endpoint reported a 65% overall response rate per modified International Working Group Myeloproliferative Neoplasms Research and Treatment / European Competence Network on Mastocytosis (mIWG-MRT-ECNM) criteria as of the March 31, 2026 data cutoff, including 57% of patients achieving complete response (CR), CR with partial hematologic recovery (CRh), or partial response (PR) as best response. Cogent had earlier received FDA acceptance for the bezuclastinib NonAdvSM (non-advanced systemic mastocytosis) NDA in March 2026 supported by the SUMMIT Phase 3 trial. Bezuclastinib would compete against Blueprint Medicines' Ayvakit (avapritinib) in the AdvSM segment; both target KIT D816V but Cogent has emphasized bezuclastinib's cleaner off-target profile and better CNS penetration profile.
The Ultragenyx (NASDAQ: RARE) UX111 (rebisufligene etisparvovec, AAV9-delivered gene therapy for the SGSH gene) FDA PDUFA action date for Sanfilippo Syndrome Type A (mucopolysaccharidosis type IIIA, or MPS IIIA — a rare autosomal recessive lysosomal storage disorder causing progressive irreversible neurodegeneration in young children with a median life expectancy in the mid-teens) is set for Saturday September 19, 2026 (weekend PDUFA date; the FDA typically issues weekend action-date decisions on the adjacent Friday or the following Monday), five days from the September 14 opening of Morgan Stanley conference week. The resubmitted Biologics License Application under Accelerated Approval was accepted by the FDA in April 2026 following a July 2025 complete response letter that identified chemistry, manufacturing, and controls (CMC) plus manufacturing-site inspection issues. The clinical package includes up to 8 years of follow-up in treated children, with sustained cerebrospinal fluid heparan sulfate (the accumulated substrate) reduction plus preserved developmental trajectories relative to untreated natural history. If approved, UX111 would become the first-ever therapy for MPS IIIA anywhere in the world; the drug would also confer a Rare Pediatric Disease Priority Review Voucher on Ultragenyx (historically valued $150-350 million on the secondary market). Manufacturing runs domestically at Andelyn Biosciences in Columbus, Ohio and Ultragenyx's own Bedford, Massachusetts facility.
Pharming Group N.V. (NASDAQ: PHAR) announced Friday September 11, 2026 that the FDA approved the company's supplemental new drug application (sNDA) for Joenja (leniolisib, an oral selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor) at 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg with activated phosphoinositide 3-kinase delta syndrome (APDS). Joenja becomes the first FDA-approved treatment for children aged 4-11 with APDS, a rare primary immunodeficiency driven by gain-of-function mutations in the PIK3CD or PIK3R1 genes producing constitutive PI3Kδ pathway activation and recurrent sinopulmonary infections. Approval follows the February 2026 complete response letter, which required additional supportive data. The newly approved doses will be available through Pharming's specialty distribution network and patient-support infrastructure starting October 2026. Joenja was originally approved in March 2023 for APDS patients aged 12 and older; the pediatric label expansion extends the treated population substantially.
Telix Pharmaceuticals Limited (NASDAQ: TLX) reached its FDA Prescription Drug User Fee Act (PDUFA) action date of Friday September 11, 2026 for TLX101-Px (Pixclara, floretyrosine F 18, also called 18F-FET), an investigational positron emission tomography (PET) imaging agent designed to help distinguish recurrent or progressive glioma from treatment-related changes in adult and pediatric patients with previously treated glioma. The FDA had accepted the resubmitted NDA in April 2026 following an earlier complete response letter. Pixclara carries FDA Fast Track and Orphan Drug designations. Standard MRI post-treatment surveillance struggles to differentiate true tumor progression from pseudoprogression or radiation necrosis; a PET imaging agent that binds selectively to actively metabolizing tumor cells addresses a substantial diagnostic gap. If approved, Pixclara would be one of the first tumor-specific glioma PET agents to enter the U.S. market. The company had not confirmed the FDA action outcome as of end of Friday U.S. business hours; investors were watching for a Monday morning announcement.
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced Thursday September 10, 2026 that the FDA granted Priority Review to a supplemental Biologics License Application (sBLA) for Enspryng (satralizumab, a humanized anti-interleukin-6 receptor monoclonal antibody using recycling-antibody technology for extended IL-6 receptor blockade) for the treatment of myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). MOGAD is a rare inflammatory central nervous system disorder attacking optic nerves, brain, and spinal cord with estimated prevalence of 0.51 to 3.42 per 100,000 people. It has no FDA-approved disease-modifying treatments. The Phase 3 METEOROID trial met its primary endpoint of time to first relapse during double-blind treatment with a 68% reduction in relapse risk versus placebo (p=0.0025) and secondary endpoints including 87% of Enspryng-treated patients relapse-free at 48 weeks (versus 67% on placebo), 66% lower annualized relapse rate, 79% fewer active MRI lesions, and 73% reduced rescue-therapy use. The FDA action date is January 10, 2027. Enspryng is currently approved in approximately 90 countries for neuromyelitis optica spectrum disorder (NMOSD).
As of Tuesday September 8, 2026, the FDA has issued no proposed rule, no Federal Register notice, and no interim enforcement policy following the July 23-24, 2026 Pharmacy Compounding Advisory Committee (PCAC) 5-3 vote recommending six of seven candidate peptides — BPC-157, KPV, TB-500, MOTS-c, Semax, and Epitalon — for inclusion on the Section 503A Bulk Drug Substances List; emideltide (DSIP) was rejected. Forty-six days have passed since the meeting ended on July 24. Center for Drug Evaluation and Research warning-letter activity to compounding pharmacies has remained elevated through Q3 2026, and no acting-FDA-commissioner statement has updated the industry timeline. The February 2027 PCAC meeting to consider cathelicidin (LL-37), GHK-Cu, dihexa acetate, melanotan II, and PEG-MGF remains scheduled. Nominated FDA Commissioner Dr. Heidi Overton (August 19, 2026) continues to await Senate confirmation with Kyle Diamantas leading the agency in the interim. Formal FDA rulemaking to translate the PCAC recommendation into a proposed rule and then a final rule typically runs 12 to 24 months from PCAC vote; the interval of 46 days is within the normal window but longer than industry advocates had modeled at the meeting.
A factual correction on the Rare Pediatric Disease Priority Review Voucher (PRV) program timeline: the Consolidated Appropriations Act 2026, signed by President Trump on February 3, 2026, reauthorized the RPD PRV program through September 2029, materially reducing the sunset risk that had shadowed rare-disease commercial modeling. The reauthorization was passed via the Mikaela Naylon Give Kids a Chance Act embedded in the CAA and covers all rare pediatric disease sponsors submitting NDAs and BLAs during the reauthorization window. Since program launch in 2012, 63 RPD PRVs have been awarded across 47 rare diseases from Duchenne muscular dystrophy to hemophilia A. Recent PRV secondary-market pricing has ranged from $67 million (2015 low) to $350 million (2015 high), with recent transactions clustered in the $100-250 million range. Arrowhead sold a PRV for $215 million alongside the plozasiran FCS approval earlier in 2026, and Ionis Pharmaceuticals received a PRV alongside the September 3 Zanvastro (zilganersen) Alexander disease approval. The reauthorization also amends the Orphan Drug Act to clarify that orphan drug exclusivity applies to the FDA's approved use or indication within a rare disease rather than the entire rare disease.