FDA issued a warning letter to Fresenius Kabi AG CEO Pierluigi Antonelli on September 22, 2026, citing CGMP violations found during an inspection of Fresenius Kabi USA's sterile injectable plant in Melrose Park, Illinois, from January 27 to February 18, 2026. Violations include inadequate investigations of endotoxin test failures tied to adverse events with famotidine for injection, leading to a recall of three batches, and repeated media fill failures on aseptic lines in 2024 and 2025. FDA found the firm's March 11, 2026 response inadequate and warned that failure to correct the violations could bring seizure or injunction.
The FDA approved Ultragenyx's Fayuvi for Sanfilippo syndrome type A, the first-ever treatment for that condition, shortly after approving the company's first gene therapy, Genglycos, for glycogen storage disease type Ia; each approval came with a priority review voucher worth as much as $200 million. The wins follow the Phase 3 Asire trial failure of apazunersen in Angelman syndrome, which cut the stock nearly 50%, and a January failure of setrusumab in osteogenesis imperfecta that erased $1 billion in value. CEO Emil Kakkis says Ultragenyx will implement significant expense reductions while keeping the setrusumab program and awaiting an upcoming Angelman readout.
The FDA has approved tavapadon, branded Juvmo, for adults with Parkinson's disease, giving AbbVie a marketed asset from its $8.7 billion Cerevel Therapeutics acquisition. BMO calls Juvmo the first selective D1/D5 receptor agonist available in the U.S., usable with or without levodopa. AbbVie will list it at $4,900 per 30-day supply, or $58,800 per year, and expects U.S. availability next month, targeting a market opportunity the company puts at $5 billion.
Two nonprofits are launching their gene therapy Waskyra, which won FDA approval at the end of last year, with a list price of $3.25 million. The therapy treats a rare immune disease. Further details of the launch are behind the paywall.
The FDA has cleared Egetis Therapeutics' Emcitate, a drug for a rare X-linked genetic disease that shortens life expectancy to 35 years. Egetis secured the US approval on Monday. Next steps for the company's commercialization of Emcitate were not disclosed.
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.
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 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.
China now accounts for 82% of the 140 in vivo CAR-T-cell therapies in development globally, according to an analysis by John Wu at Boston Consulting Group and colleagues published last month. Most foundational preclinical work came from the United States and Germany, but China has moved fastest to the clinic, aided by government support and investigator-initiated trials that bypass drug-regulator oversight. The FDA this month approved the first China-developed in vivo CAR-T-cell therapy, for blood cancers, for clinical testing.
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.
At the 2026 SOHO annual meeting, Jasmine M. Zain of Memorial Sloan Kettering described how to attribute secondary T-cell malignancies to CAR-T therapy, requiring clonality flow cytometry, PCR detection of the CAR vector in the tumor genome, and insertion-site analysis relative to proto-oncogene or tumor suppressor promoters, with a theoretical risk window of 300 base pairs. David Sallman covered lower-risk MDS updates including luspatercept for ring sideroblast patients, the recent FDA approval of imetelstat, and eprenetapopt plus azacitidine data in TP53-mutated higher-risk MDS. Julie M. Vose reviewed the CLL landscape, from first-generation ibrutinib to better-tolerated second-generation BTK inhibitors acalabrutinib and zanubrutinib, approved pirtobrutinib in relapsed or refractory disease, and bispecific CD20xCD3 antibodies and lisocabtagene maraleucel under study.
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.
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.
