The FDA approved the Autus Size-Adjustable Valve, developed by Autus Valve Technologies and approved via Edwards Lifesciences, for pediatric patients with congenital pulmonary valve disease. It is the first heart valve that can be expanded after implantation, growing from about 13 millimeters to up to 22 millimeters, and the first U.S.-approved valve with polymeric rather than animal-derived leaflets. Approval rests on a study of 62 pediatric patients at 12 U.S. sites, with no deaths or clot-related complications; longer-term studies are under way.
Kyverna has started a rolling BLA submission to the FDA for miv-cel and plans to complete it in the fourth quarter of this year. If approved, miv-cel would become the first CAR-T therapy authorized for an autoimmune disease, moving CAR-T beyond oncology for the first time since its commercialization more than a decade ago.
FDA placed Chengdu Shengnuo Pharmaceutical, a wholly owned subsidiary of the Shanghai STAR-listed peptide maker Shengnuo Biology, on Import Alert 66-40 on September 19, without first issuing a warning letter, meaning its products can be detained at US customs without inspection. The company disclosed the move on September 22. The action comes as US federal enforcers penalize a large Houston compounding pharmacy for GLP-1 weight-loss drugs and attorneys general from 20 states jointly press for tighter controls on GLP-1 compounders.
The FDA has approved AbbVie's JUVO (tavapadon) for adults with Parkinson's disease, announced on September 28, 2026. JUVO is the first and only approved selective D1/D5 receptor agonist and is taken once daily. The approval marks the first new-mechanism dopaminergic therapy for Parkinson's disease in decades.
China's NMPA has approved Bristol Myers Squibb's iberdomide capsules (brand name Zanfu) via priority review on September 30, 2026, in combination with daratumumab and dexamethasone for adults with multiple myeloma who received at least one prior line including a proteasome inhibitor and an immunomodulatory drug. Iberdomide is the first approved Cereblon E3 ubiquitin ligase modulator (CELMoD) and BMS's first marketed product from its targeted protein degradation work.
The 2026 edition of the CSCO primary liver cancer guideline has raised yttrium-90 microsphere SIRT (selective internal radiation therapy) to a Grade I, Category 1A recommendation. At the CSCO annual meeting's nuclear medicine session, Professors Yang Aimin of the First Affiliated Hospital of Xi'an Jiaotong University and Jia Weidong of the First Affiliated Hospital of USTC presented a new Chinese expert consensus on whole-process nuclear medicine management of Y-90 SIRT for liver malignancies. Jia Weidong discussed SIRT as a new option in conversion therapy for liver cancer.
The FDA has expanded Bristol Myers Squibb's Camzyos (mavacamten) indication to cover symptomatic obstructive hypertrophic cardiomyopathy in adults and paediatric patients weighing at least 30kg. The decision rests on the Phase III SCOUT-HCM trial in 44 adolescents aged 12 to under 18, where the cardiac myosin inhibitor significantly reduced Valsalva LVOT gradient at week 28 versus placebo with no new safety concerns. Camzyos, first approved in 2022 and now cleared in more than 60 countries, is in discussions with other regulators for paediatric approvals.
China's NMPA has approved Merck's subcutaneous formulation of the PD-1 inhibitor pembrolizumab, covering 19 solid tumour indications, making it the first and currently only subcutaneous PD-1 inhibitor approved in China. The approval, announced on 29 September 2026, is based on data from study MK-3475A-D77. The approval covers the same indication set as the existing intravenous formulation.
The FDA has approved a new drug for pancreatic cancer that reportedly extends survival in patients. The approval was reported by Scientific American on August 26 and relayed by Chinese industry media on August 29. No drug name, trial details or dosing information were given in the report.
Edwards' EVOQUE transcatheter tricuspid valve replacement system, the first such system to hold both CE marking and FDA approval, has received special access in China and completed its first clinical uses at Beijing's Fuwai Hospital. The system offers an interventional option for patients with severe tricuspid regurgitation, including elderly patients with multiple comorbidities or high surgical risk. Broader use in Chinese patients is expected to follow.
The FDA approved AbbVie's JUVMO (tavapadon) on September 25, 2026 for the treatment of Parkinson's disease in adults. JUVMO is the first and currently only selective D1/D5 receptor agonist approved for adult Parkinson's disease, and the article describes it as the first breakthrough in dopaminergic therapy for Parkinson's in decades.
FDA issued a warning letter, reference 320-26-129, to Fresenius Kabi USA CEO Pierluigi Antonelli on September 22, 2026, citing CGMP violations found at its sterile injectable manufacturing facility in Melrose Park, Illinois, inspected from January 27 to February 18, 2026. FDA says the firm failed to adequately investigate drug product failures, including bioburden action limit failures linked to adverse events with famotidine for injection, leading to a recall of three famotidine injection batches after an endotoxin failure in retained samples. FDA found the company's March 11, 2026 response inadequate and warned that failure to correct violations may result in seizure or injunction.
The FDA approved Camzyos (mavacamten) as the first treatment to improve functional capacity and symptoms in children with symptomatic obstructive hypertrophic cardiomyopathy, extending approval to pediatric patients weighing at least 30 kg, after adult approval in 2022. Efficacy was supported by the phase 3 SCOUT-HCM trial in 44 patients aged 12-18, where once-daily dosing for 28 weeks significantly reduced the Valsalva LVOT gradient versus placebo. The drug carries a boxed warning for heart failure risk and is available only through the Camzyos REMS program.
The FDA has accepted Roche's approval application for fenebrutinib, an experimental multiple sclerosis pill, under a priority review with a six-month timeline, covering both relapsed and primary progressive forms of the disease. The application rests on three late-stage studies that met their main objectives, but approval chances could be complicated by an imbalance in deaths, eight among fenebrutinib recipients versus one on Aubagio in two Phase 3 trials, and a case of liver enzyme elevations meeting Hy's law. Roche did not say when a decision might come.
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.
Merck and Daiichi Sankyo have voluntarily withdrawn their accelerated approval application for the antibody-drug conjugate ifinatamab deruxtecan (I-DXd) in extensive-stage small cell lung cancer after platinum-based chemotherapy, after the FDA said the data did not satisfy requirements for the proposed indication. The application rested on a 48.2% confirmed objective response rate from the Phase 2 IDeate-Lung01 study. A Phase 3 trial in ES-SCLC with enrollment nearing completion continues and could support a future filing.
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.