ClinicaSpace

Cancer cocktails pairing Moderna’s mRNA-4359 with Merck’s Keytruda and Marengo’s invikafusp alfa with Gilead Sciences’ Trodelvy showed promising results, while a complex combination by Agenus and MiNK Therapeutics failed to elicit an overall response.

As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.

The FDA is signaling change, but actual success depends on more than simply bringing in a new leader at the Center for Biologics Evaluation and Research; it requires accountability, transparency and consistent action.

Approved Thursday via the FDA’s Commissioner’s National Priority Voucher program, Otarmeni is the first gene therapy for hearing loss—and the first treatment to target an underlying cause of the condition.

With a greenlight for ibogaine to enter clinical testing and three unnamed products set to receive Commissioner’s National Priority Vouchers this week, it’s full speed ahead for psychedelics. But will sidestepping normal regulatory protocols actually be a net negative for the field?

After Replimune’s advanced melanoma drug was rejected for a second time, CEO Sushil Patel slammed the FDA for failing to exercise regulatory flexibility, while other experts bemoaned the agency’s lack of consistency. With new safety guidelines for gene editing therapies, the FDA has taken a first step toward fixing both problems.

The Merck update, which will shed light on a $588 million bet to succeed Keytruda, is part of a roster of presentations that could shape the future of ADCs, protein degraders and KRAS-targeted therapies.

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.