ClinicaSpace

With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA.

William Blair hailed a positive readout in cutaneous lupus erythematosus as a turning point for Biogen, while RBC Capital analysts called the results “another derisking step” for the company’s immunology and inflammation pipeline.

Accumulating scientific evidence and industry interest from Eli Lilly, Altimmune and startup Baseline Therapeutics is driving further research on the therapeutic potential of GLP-1 receptor agonists in treating substance use disorders.

Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.

Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential billion-dollar market and banking on local delivery and the small amount of drug required to overcome key safety concerns.

The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher.

A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.

As Congress debates renewing the Act for ALS, it must prepare for the coming era of precision ALS medicine by prioritizing early-stage research to more quickly bring effective treatments to the market.

Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a need for more rigorous evidence.

The Hunter syndrome space suffered a setback in February when the FDA turned down REGENXBIO’s investigational gene therapy, raising urgent questions about whether competitor Denali Therapeutics can clear the agency’s bar next month.