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After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination of 20 years of hard work unraveling the mysteries of the blood-brain barrier.
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At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.
Doubling survival in pancreatic cancer, a long-fought rare disease approval, a massive IPO and ambitious biotech entrepreneurs have BioSpace Senior Editor Annalee Armstrong feeling upbeat about the biotech scene.
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.
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Novo Nordisk’s oral Wegovy has a few months’ head start on Eli Lilly’s newly approved pill. While the Indianapolis pharma has come from behind the Danish rival in the weight loss space before, last time it clearly had the better drug.
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The framework, first introduced by FDA Commissioner Marty Makary and Center for Biologics Evaluation and Research head Vinay Prasad in November, was criticized for lacking detailed guidance. Agency leaders elucidated on the pathway for personalized medicines on Monday.
The new structure will help Merck as it slides toward a loss of exclusivity for Keytruda, pharma’s best-selling drug.
Ralph Abraham, a vocal vaccine skeptic who served at the agency for just three months, has stepped down due to “unforeseen family obligations,” according to the CDC.
Bysanti is based on iloperidone, an active metabolite of a compound that forms the core of Fanapt, another drug by Vanda Pharmaceuticals.
A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent was invalid because its elements were naturally occurring. The appeals court on Friday said that this original decision adopts a “narrow” view of the invention.
The centerpiece of the takeover is anito-cel, a CAR T therapy under development for relapsed or refractory multiple myeloma. An FDA decision on the therapy is expected by December 2026.
Head-to-head trials are often reserved for approved therapies as drugmakers try to one-up each other to gain further market share. In this case, Novo Nordisk ran the test at the candidate stage, and the bet did not pay off.
The U.S. Congress greenlit a historic $315 million in federal ALS research funding for 2026 amid Rare Disease Month, spotlighting biotech progress like VectorY Therapeutics’ first patient dosing in its TDP-43-targeting PIONEER-ALS trial and EverythingALS’ pharma consortia driving biomarker innovations and trial alignment.
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.