Biologics

FDA Probes Malignancy Risk Linked to Zolgensma Gene Therapy

FDA is investigating the safety of Novartis’ Zolgensma, an adeno-associated virus (AAV) vector-based gene therapy for spinal muscular atrophy (SMA), based on postmarketing adverse event reports of malignancies in treated patients. The adverse events include confirmed reports of “product-related insertional oncogenesis involving the GLI1 (Glioma-Associated Oncogene Homolog 1) gene,” FDA says. “GLI1 is an oncogene that has the potential to cause cancer when overexpressed or abnormally activated.”

FDA’s probe follows reports last week that Novartis had disclosed nine reports of tumors in children treated with Zolgensma (see earlier story).

The agency said reported tumors have involved the central and peripheral nervous systems, spinal and paraspinal regions, and kidneys.

FDA determined that the risk of neoplasia, including malignancy, also applies to Novartis' Itvisma because the two SMA gene therapies contain the same vector and construct. No tumors have been reported following Itvisma treatment.

Zolgensma was approved in May 2019 for children younger than 2 years with SMA, while Itvisma was approved in November 2025 for patients ages 2 years and older.

FDA Orders Eye Safety Warnings for All GLP-1 Drugs

FDA is requiring manufacturers of all glucagon-like peptide-1 receptor agonist (GLP-1 RA) drugs to add warnings about a potential increased risk of non-arteritic anterior ischemic optic neuropathy (NAION), a rare eye condition that can cause sudden, permanent vision loss. NAION typically causes acute, painless vision loss

FDA Posts 18 Form 483 Inspection Records

FDA has posted 20 Form 483 inspection records (see below), including reports involving Novo Nordisk, AnaptysBio, Protagonist Therapeutics, Ascendis Pharma and Charles River Laboratories, as the agency continues expanding public access to inspection findings through its electronic reading room. Their availability is part of the agency’s expanded transparency initiative

Bias in Extension Studies Despite Their Value to FDA Safety Reviews: JAMA

Long-term clinical trial extension studies can provide important evidence about drug safety and durability of treatment effects, but their findings may be compromised by selection bias, patient attrition and the loss of randomized comparison groups, according to a 10/7 JAMA editorial. The editorial highlights a challenge for FDA

Ex-HHS Aide Says Overton Pressured Makary Over Vaccine Approval

Former senior HHS adviser Calley Means has accused FDA commissioner nominee Heidi Overton of pressuring former commissioner Marty Makary to approve Moderna's mRNA influenza vaccine despite his scientific objections, alleging that she conditioned assistance on other policy matters on his willingness to approve the product. Means resigned 10/

Read more

FDA Orders Eye Safety Warnings for All GLP-1 Drugs

FDA Posts 18 Form 483 Inspection Records

Bias in Extension Studies Despite Their Value to FDA Safety Reviews: JAMA

Ex-HHS Aide Says Overton Pressured Makary Over Vaccine Approval