FDA issued Warning Letter 320-26-44 to VeganicSKN Limited, a drug manufacturer in Rocklea, Queensland, Australia, following an inspection from July 15 to 21, 2025, citing significant CGMP violations that render its drug products adulterated. The letter cites an out-of-specification investigation opened on December 4, 2024, after a contract lab reported a failing result on August 28, 2024, with the batch still released and shipped to the U.S. market and the investigation closed on July 11, 2025 without a root cause. FDA found the August 11, 2025 response inadequate and requires a retrospective review of invalidated OOS results plus a CAPA and remediation plan for its testing and supplier-verification systems.
The FDA issued a warning letter, reference 320-26-127 dated September 18, 2026, to Curia New York, Inc. CEO Philip Macnabb after a January 28 to February 5, 2026 inspection of its API manufacturing facility in Rensselaer, New York, found CGMP deviations. Analysts performed thousands of unexplained HPLC and UPLC trial injections over 30 months using Empower, over 70 in October 2025 alone via TotalChrom, and about 20 percent of Empower trial injections were acquired or altered in "Developer" mode. The FDA found the firm's February 27, 2026 response inadequate and requires a comprehensive independent investigation of all trial injections.
The FDA approved Emcitate (tiratricol) oral suspension, from Egetis Therapeutics US Inc., for peripheral thyrotoxicosis in patients with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. It is the first FDA-approved therapy for the rare genetic disease, which mainly affects males. Evidence came from a randomized placebo-controlled trial (NCT05579327) and a longer-term open-label study spanning infants to adults. The once-daily liquid can be given by mouth or feeding tube.
The FDA approved Mirum Pharmaceuticals' zilurgisertib, to be marketed as Atebrioz, for fibrodysplasia ossificans progressiva in patients 12 years and older, making it the third FOP treatment after Ipsen's Sohonos (August 2023) and Regeneron's Pasatru. The once-daily ALK2 inhibitor, which Mirum gained full ownership of in April after licensing from Incyte, showed an 81% reduction in new lesion volume and 99% reduction in total volume versus placebo at 24 weeks in the Phase 2 PROGRESS study, with effects maintained through week 48. Mirum is now conducting trials in children as young as 2 years.
Merck & Co. and Daiichi Sankyo withdrew their U.S. application for accelerated approval of ifinatamab deruxtecan, or I-DXd, in extensive-stage small-cell lung cancer after the FDA indicated Phase 2 response rate data were insufficient; a decision had been expected by Oct. 10. The B7-H3-targeting drug is still being tested against chemotherapy in relapsed disease, a trial in which recruitment was temporarily halted last year after higher than anticipated deaths from a serious lung condition. Competing B7-H3 drugs licensed to GSK (riz-rez) and Roche (tam-peli) have advanced, and I-DXd is also in Phase 3 testing in prostate cancer and esophageal squamous cell carcinoma.
The US FDA has approved belzutifan combined with lenvatinib, from Merck and Eisai, for adults with advanced clear cell renal cell carcinoma previously treated with a PD-1/PD-L1 inhibitor. The companies say it is the first approved regimen pairing a HIF-2α inhibitor with a multi-target VEGFR-TKI in this setting. Belzutifan, developed by Merck, was the first approved HIF-2α inhibitor and blocks the HIF-2α signalling pathway.
Japan has approved the world's first regenerative medicine product derived from induced pluripotent stem cells (iPSC) for treating Parkinson's disease. The milestone comes two decades after Shinya Yamanaka reprogrammed fibroblasts back to a blank state in 2006, with iPSC-derived cells now being implanted into patients' brains. Parkinson's becomes the first neurological field where the technology has broken through.
The FDA approved nipocalimab on August 25 for adults and adolescents aged 12 and older with warm autoimmune hemolytic anemia who are receiving or have received corticosteroid treatment, making it the first approved therapy in this indication. Nipocalimab is an immunoselective neonatal Fc receptor blocker that lowers circulating IgG, including the autoantibodies driving wAIHA. Its pivotal phase II/III ENERGY trial, a 24-week randomized double-blind study published in Blood, evaluated efficacy and safety in these patients.
The European Commission's Directorate-General for Health and Food Safety has withdrawn the EU marketing authorisation for Hansoh Pharmaceutical's aumolertinib tablets, marketed overseas as Aumseqa. The decision, dated September 25, concerns the use of osimertinib clinical data in the marketing application. The drug had been approved on February 12, 2026, less than eight months earlier, and Hansoh says it will resubmit the application as soon as possible.
The FDA approved Rasonque (daraxonrasib) on August 26, 2026 for metastatic pancreatic adenocarcinoma in patients previously treated with at least one systemic therapy. It is the first direct RAS-targeted oral drug approved in pancreatic cancer, a disease where more than 90% of patients carry KRAS mutations. The approval comes 44 years after RAS was identified as the first human oncogene in 1982.
On September 23, an FDA advisory panel of 10 experts voted 6-4 on Grail's multi-cancer early detection test Galleri. Galleri has been offered in the US as an LDT since mid-2021, with nearly 600,000 tests sold by the end of June 2026 and screening revenue of $42.6 million in the second quarter of 2026 alone. The panel's divided vote precedes further FDA consideration of the test.
The FDA has approved AbbVie's tavapadon, a next-generation therapy for Parkinson's disease designed to provide symptom relief comparable to its predecessors with fewer side effects. No further details on dosing, launch timing or pricing were available. The approval gives Parkinson's clinicians a new dopamine-based treatment option.
The FDA has approved Eli Lilly's Onswik, a once-weekly basal insulin for adults with type 2 diabetes. The approval gives patients an alternative to daily long-acting insulin injections. Details beyond the approval itself are available to STAT+ subscribers.
The FDA has approved Eli Lilly's once-weekly basal insulin Efsitora alfa (brand name Onswik), announced on September 24, 2026, to improve glycaemic control in adults with type 2 diabetes alongside diet and exercise. Efsitora is a fusion protein linking a novel single-chain insulin variant to a human IgG2-Fc domain, extending half-life to allow weekly subcutaneous injection instead of daily dosing.
Lilly's once-weekly insulin Efsitora alfa has been approved for marketing in China. Efsitora alfa is an Fc-fusion single-chain insulin of 299 amino acids expressed in CHO cells, with 24 disulfide bonds and a molecular weight of 64 kDa. In the trials QWINT-1 and QWINT-2 it showed glucose-lowering effects non-inferior to insulin glargine and insulin degludec, respectively.
The FDA has approved Eli Lilly's once-weekly long-acting basal insulin Onswik (insulin efsitora alfa) for adults with type 2 diabetes, offering an alternative to daily insulin injections. The approval reflects a broader effort across insulin and GLP-1 drugs to extend dosing intervals for long-term treatment. Onswik now becomes a new treatment option for adult patients in the United States.
The FDA sent Empower Clinic Services, which does business as Empower Pharmacy, a warning letter dated Sept. 18 citing insanitary conditions at a Houston site inspected in November 2025 and evidence that its compounded tirzepatide and semaglutide products are essentially copies of FDA-approved drugs in violation of Section 503A. As of Sept. 24, Empower still listed both products for sale. The company has 15 working days to respond in writing with steps taken to correct and prevent the violations.
An FDA expert committee voted on September 23 that the clinical benefits of Galleri, a multi-cancer early-detection blood test, outweigh its risks. The vote marks a key step toward formal FDA review of the product, which is positioned as a blood draw for early cancer screening. The agency's decision process now moves forward following the panel's endorsement.
On September 21, Eli Lilly said China's NMPA approved an update to the label for Mufengda (tirzepatide injection), its GIP/GLP-1 dual-target weekly injection, adding clinical study data in adults with heart failure with preserved ejection fraction and obesity. The update incorporates SUMMIT study results showing a 38% reduction in the risk of worsening in these patients. The drug was previously positioned for glucose lowering and weight reduction.
FDA advisors have voted to recommend Grail's multi-cancer blood test Galleri, despite a major trial setback earlier this year and ongoing questions over its efficacy. Grail now awaits an FDA decision on the test. The outcome leaves the company positioned for a potential approval ahead.