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Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace,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
The discovery of a tumor in a patient who received REGENXBIO’s gene therapy for Hurler syndrome prompted the FDA to place a hold on that program along with the company’s Hunter syndrome program, which is awaiting an FDA decision on or before Feb. 8.
Darzalex Faspro, in combination with an anti-cancer triplet, is the first anti-CD38-based regimen for newly diagnosed patients with multiple myeloma, regardless of eligibility for stem cell transplantation.
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
True inspection readiness is about the integrity of a company’s entire system.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
The U.S. regulator shared the roadmap for implementing the program, first proposed in August 2025, and teased changes made in response to industry feedback.
The initiative could tackle the first-mover disadvantage some CDMOs believe deters early customers, but leaders at companies including Novo Nordisk see hurdles to implementing the changes.
As biotechs faced investors at the J.P. Morgan Healthcare Conference this week, they emphasized agreement with the FDA on clinical trial design and regulatory pathways to approval. Atara, meanwhile, lamented the agency’s “complete reversal of position” after its therapy for a rare surgical complication was rejected.