The FDA is dominating the headlines once again thisweek.
Days after FDA Commissioner Marty Makary appeared to question uniQure’s gene therapy candidate for Huntington’s disease, the company revealed that the agency will require it to conduct a randomized, double-blind, sham surgery–controlled Phase 3 study. The FDA also published anothercomplete response letter (CRL), this one for REGENXBIO’s gene therapy for Hunter syndrome. The rejection, sustained by the biotech early last month, was driven by issues with the study’s population, controls and use of surrogate markers to measure efficacy, according to the document.
Meanwhile, regulatory experts have expressed concernsthat the FDA’s circle of trust is shrinking, making many decisions feel like “fiat”—both in terms of individual drug applications and policy. The FDA has reportedly initiated a probe into complaints that a toxic workplace is fostered by CBER director Vinay Prasad, who is at the heart of many of these decisions. Finally, the biopharma industry continues to react to the agency’s pivot from a requirement of two pivotal trials to one for approval, asking why now, what are the risks and what exactly the FDA expects from this one trial.
Still on the gene therapy front, Sarepta Therapeutics CEO Doug Ingram stepped down last week to spend more time with family as the company’s muscular dystrophy mission hits home. Also during the company’s fourth quarter earnings call, Sarepta projected that sales of its embattled Duchenne muscular dystrophy gene therapy Elevidys will be flat or down as far as 15% in 2026.
On the obesity front, Eli Lilly topped Novo Nordisk again in a weight loss trial, this time in a Lilly-sponsored study of patients with type 2 diabetes. But don’t count Novo out yet. The company is actively seeking out new obesity assets, according to business development executive Tamara Darsow. Just last week, Novo linked with Boston’sVivtex to advance novel weight loss pills.
Finally, check out BioPham Executive this week for a rundown of 2025’s top-selling assets—spoiler: Merck’s Keytruda held onto its crown as number one—and a story on former2seventy exec Chip Baird’s new role as CEO of recently launched Poplar Therapeutics, which secured a $45 million series A extension this week.
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