The FDA has approved Ultragenyx's gene therapy Fayuvi for a rare neurodegenerative disorder, which the source calls a first-in-disease gene therapy. The source notes the approval is a belated regulatory win for Ultragenyx after an earlier rejection and a recent clinical setback.
Why it matters: FDA approval of a first-in-disease gene therapy reverses an earlier rejection and offsets a recent clinical setback, giving Ultragenyx a fresh start.
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy from Ultragenyx Pharmaceutical, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy.
Bayer's Kerendia has received FDA approval for chronic kidney disease associated with type 1 diabetes in adults, making it the first new drug approved for this indication in nearly 30 years. The original article describes the drug as positioned as a cardio-renal therapy, and the approval ends three decades without a new drug in this field.
The FDA has approved Bayer's Kerendia (finerenone) for adults with chronic kidney disease associated with type 1 diabetes. Bayer said on September 17 that this is the first fundamentally new treatment option the FDA has approved for T1D-related CKD in more than 30 years. The agency accepted Bayer's supplemental new drug application for the indication in May and granted it priority review.
The FDA has approved Bayer's Kerendia (finerenone) for adults with chronic kidney disease associated with type 1 diabetes, Bayer announced on September 17, 2026. It is the first drug approved by the FDA for this indication in more than 30 years. The approval fills a long-standing treatment gap for CKD patients with type 1 diabetes.
Bayer's sevapritinib received FDA accelerated approval as first-line treatment for adults with locally advanced or metastatic non-squamous non-small cell lung cancer harboring HER2 tyrosine kinase domain activating mutations. The approval extends the drug's use from its second-line approval in November 2025, allowing oral targeted therapy from diagnosis. In the SOHO-01 trial, the first-line cohort of 69 untreated patients showed a confirmed ORR of 75%, with 6% complete and 70% partial responses.
At the 2026 ESC Congress, HighTide Therapeutics' oral AMPK-NLRP3 dual-mechanism drug HTD1801 showed multi-organ effects across cardiovascular-kidney-metabolic (CKM) disease. In two phase 3 trials in type 2 diabetes it met primary endpoints, with HbA1c changes of -1.3% and -1.2% at week 24, sustained to week 52. It also lowered LDL-C and non-HDL-C, and in a glomerular hyperfiltration subgroup reduced the eGFR slope by 3.00 mL/min/1.73 m2 per year versus placebo.
On September 15, 2026, the FDA published the final design of its Expedited IND Pilot, which pairs selected sponsors with Qualified Research Institutions to submit IND components on a rolling basis during Pre-IND review. The agency explicitly cited China and Australia, noting FIH trials in the US can take up to two years while both countries move faster; HHS data cited show China's share of global Phase 1 trials passed the US in 2021, with over 7,100 registered trials in 2024, about 39% of the global total. The 30-day statutory review window and Clinical Hold authority are unchanged; FDA estimates trimming unnecessary submission content alone could save 6 to 12 months, and pilot experience may inform a formal QRI qualification system.
On August 25, 2026, the FDA approved the HER2 bispecific antibody zanidatamab for first-line treatment of adults with unresectable locally advanced or metastatic HER2-positive gastric, gastroesophageal junction or esophageal adenocarcinoma confirmed by an FDA-approved test. One approval covers zanidatamab combined with tislelizumab plus fluoropyrimidine and platinum chemotherapy for patients with HER2-positive disease defined as IHC 3+ or IHC 2+/ISH+. The approvals were based on the HERIZON-GEA-01 study.
On September 14, 2026, Curium announced FDA full approval of Bexlutry (oxodtreotide), the first approved generic radioligand therapy, termed a "radioligand equivalent." It copies Novartis's Lutathera, an SSTR2-targeted radioligand therapy obtained through Novartis's acquisition of Advanced Accelerator Applications, first FDA-approved in 2018 for gastroenteropancreatic neuroendocrine tumors, with global sales of $816 million in 2025.
FDA is signaling tighter scrutiny of early-stage clinical data generated in China while stopping short of the outright ban sought by some members of Congress. Reps. John Moolenaar and Ben Cline asked Acting Commissioner Kyle Diamantas in an Aug. 20 letter to reject data from Chinese sites not inspected within the previous 12 months, a requirement that would effectively ban such data given FDA's inspection manpower. The agency must balance congressional pressure against multinational biopharmas' reliance on China in global drug development.
The FDA has opened a pilot program for an expedited investigational new drug (IND) application process, aiming to accelerate U.S. phase 1 clinical trials. The agency wants U.S. phase 1 trial timelines to match those in China and Australia. The pilot follows an initiative to speed early-stage trials that the FDA signaled over the summer.
Final two-year Phase 3 results show Trutakna, Vera Therapeutics' drug for IgA nephropathy, stabilized kidney function and reduced the risk of kidney-related death and other complications. The FDA granted Trutakna accelerated approval for IgAN in July, and Vera plans to file for full approval in the fourth quarter.
The FDA has approved Bexlutry, Curium's lutetium Lu 177 dotatate injection, as the industry's first radioligand equivalent. The approval for the Boston company follows a court ruling that cleared Curium of all patent claims brought by Novartis, whose reference product is Lutathera.
The FDA has accepted Takeda's new drug application for Zasocitinib (TAK-279), an oral TYK2 inhibitor for adults with moderate to severe plaque psoriasis, and granted priority review, Takeda announced on September 14. The drug was originally developed by Nimbus Therapeutics and Schrödinger, and Takeda acquired it from Nimbus in 2022 for a $4 billion upfront payment in a deal worth up to $6 billion.
The FDA has approved Telix's Pixclara (floretyrosine F 18, or 18F-FET), the first FET-PET imaging drug for glioma approved in the U.S., more than a year after rejecting the application in April over a need for additional confirmatory clinical evidence. The diagnostic, used with PET to distinguish recurrent or progressive glioma from treatment-related change, is approved for adults and children one month of age and older. Gliomas account for around 80% of malignant brain tumors, with around 24,000 U.S. cases yearly.
Scholar Rock's apitegromab, brand name Isembyld, received FDA approval on September 11 as the first muscle-targeted therapy for spinal muscular atrophy. It is indicated for SMA patients aged 2 and older who are receiving SMN2-targeted therapy, and works by inhibiting myostatin production to promote muscle growth. US shipments begin within days of approval.
The FDA convened a public hearing on how psychedelic medicines should be used, monitored and studied in the real world, as the agency moves toward what would be the first approval of a psychedelic medicine. The discussion centered on access and safety questions raised by the anticipated approval.
FDA officials from CDER say the agency is prepared to approve psychedelic therapies for chronic psychiatric disorders based on 12-week efficacy data, before sponsors have settled how long effects last or when patients need redosing. The position appears in a New England Journal of Medicine article and clarifies what the agency's final guidance for psychedelic drug development, released in July, left open. Companies profiled include Compass Pathways, Cybin, Ataibeckly and Eli Lilly.
The FDA has approved Moderna's mRNA seasonal influenza vaccine mFlusiva for people aged 50-64. It is the first seasonal flu vaccine to use mRNA-based production, encoding full-length hemagglutinin from three WHO-recommended strains in lipid nanoparticles, allowing rapid updates to match circulating strains. In two clinical trials, mFlusiva outperformed GlaxoSmithKline's four-strain vaccine Fluarix in preventing infection or eliciting antibody responses.
