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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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Hernexeos is the second drug to secure an FDA approval under the agency’s priority voucher scheme, following in the footsteps of USAntibiotics’ Augmentin XR, which was granted the ticket in December 2025.
Aside from creating a toxic work environment, CBER Director Vinay Prasad has also been accused of berating his staff and retaliating against reviewers who questioned his decisions.
Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients.
The BioSpace team hit the ground running at the J.P. Morgan Healthcare Conference earlier this month to bring you the news from the streets of San Francisco.
After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.
Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.
In this episode of Denatured, you’ll be listening to Indu Navar, CEO and founder of EverythingALS and Dr. Olga Uspenskaya, chief medical officer at VectorY Therapeutics. We’ll be speaking about patient-pharma collaborations accelerating trials and hope, advances in ALS biology understanding and biomarker-driven endpoints.
While Boehringer Ingelheim hasn’t yet revealed what diseases it will go after, Sitryx’s oral drug candidate could potentially be disease-modifying for a variety of autoimmune and inflammatory conditions.
Novo Nordisk reported a loss in a head-to-head trial of CagriSema against Lilly’s Zepbound earlier this week. This time around, Lilly’s orforglipron bested Novo’s oral semaglutide in blood sugar control and weight reduction—albeit with a few extra discontinuations as compared to its rival.
The rejection of Atara Biotherapeutics’ Ebvallo in January was a “complete reversal” of the conesensus FDA reviewers had come to, according to a former agency employee, who said manufacturing problems were the only approvability barrier for the drug.