FDA
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.
FEATURED STORIES
The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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.
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.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
Having targeted Hims & Hers last year, the FDA has issued warnings to more telehealth companies over their promotion of GLP-1 drugs used to treat diabetes and obesity. In one case, compounded products were linked with multiple ER visits.
The FDA last October paused Intellia Therapeutics’ late-stage CRISPR studies after detecting life-threatening enzyme elevations in one patient, who died a few days later.
In a complete response letter published by the FDA on Monday, the agency said a resubmission for REGENXBIO’s Hunter syndrome gene therapy should provide evidence of normalized or improved biomarker levels or neurodevelopmental outcomes.
Here’s how drug developers can best approach interactions with the agency following last year’s seismic changes to its leadership, workforce and policies.
FDA decisions lack majority consensus, experts agree, possibly leading to less nuanced verdicts on new drug applications. This type of “fiat” decision-making, as multiple regulatory experts have called it, is also bleeding into the agency’s policymaking.
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
Yuviwel will compete with BioMarin’s Voxzogo. Meanwhile, BridgeBio is working to bring its own achondroplasia drug, the FGFR3 blocker infigratinib, to the market.
On the FDA’s docket this month are two decisions pushed back from 2025, including one for a rare form of obesity and another for dry eye disease.
This week’s Capitol Hill meetings come on the heels of rejections of ultra-rare disease drugs developed by Biohaven and Saol Therapeutics. Physicians and patient groups implored the FDA to expedite these treatments.
FDA Commissioner Marty Makary presented a new idea to staff this week: bonus payments for employees that complete regulatory review processes faster than expected.