Biologics

FDA Accepts Ultragenyx Resubmission for Gene Therapy

Ultragenyx Pharmaceutical has resubmitted a BLA seeking accelerated approval for UX111, an AAV9 gene therapy for the treatment of Sanfilippo syndrome Type A, a rare and fatal pediatric neurodegenerative disorder. Sanfilippo syndrome type A is described as a rare inherited lysosomal storage disorder caused by mutations in the SGSH gene, leading to rapid and progressive neurodegeneration beginning in early childhood.

The resubmission follows an agency-issued 7/2025 Complete Response Letter, and includes updated long-term clinical data as well as responses to chemistry, manufacturing and controls issues raised during the prior review. The company said it expects the FDA to take up to six months to review the application under existing regulatory guidelines.

UX111, also known as rebisufligene etisparvovec, is being developed as a one-time intravenous gene therapy designed to address the underlying enzyme deficiency that leads to the accumulation of heparan sulfate in the brain of patients with Sanfilippo syndrome type A, also known as MPS IIIA. There are currently no approved treatments for the disease.

Ultragenyx said the resubmitted application contains extended follow-up data showing durable neurologic, biochemical and clinical effects lasting up to 8.5 years in treated patients. FDA had previously acknowledged that neurodevelopmental outcomes and biomarker data supported evidence of treatment activity, according to the company.

The company is seeking accelerated approval based on an intermediate clinical endpoint supported by cerebrospinal fluid heparan sulfate measurements and additional biomarkers, an approach discussed with FDA during the previous review cycle.

Ultragenyx describes UX111 as an AAV9-based gene therapy designed to deliver a functional copy of the SGSH gene to the brain via a single intravenous infusion. The product aims to correct the underlying enzyme deficiency in patients with the condition, which leads to toxic buildup of heparan sulfate in brain cells and results in rapid neurological decline. Originally developed by Abeona Therapeutics, the program was transferred to Ultragenyx for late-stage development and commercialization.

If approved, UX111 will be manufactured entirely in the U.S., with production split between facilities in Ohio and Massachusetts.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome