ClinicaSpace

BioSpace looks back at 2025 and where the FDA is going in 2026.

The FDA has gained a reputation during the past year for being inconsistently flexible, particularly when it comes to rare diseases. Executives at Rezolute and CERo Therapeutics recently had positive interactions with the agency, in which they told BioSpace reviewers have been “collaborative” and “curious.

Since the FDA began publishing its rejections of drug approval filings in July last year, companies have become more forthcoming about the details of agency decisions in their own disclosures, according to biopharma and regulatory analysts.

The upcoming FDA decision for Replimune’s advanced melanoma drug could be a litmus test for the agency’s future regulatory decision-making, analysts say, with implications stretching well beyond one company.

While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external controls—the application of this flexibility appears to be inconsistent. One former regulator says the situation is more nuanced.

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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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.