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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.
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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.
Alternatives to opioids are desperately needed to better treat moderate to severe acute pain, but to date, we’ve seen few novel analgesics hit the market.
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Eli Lilly’s win in a head-to-head trial drove Novo Nordisk’s market cap to pre-Wegovy levels not long after the victor became the first pharma company to top a $1 trillion valuation. It seems one company can do no right, while the other can do no wrong.
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We are in an unprecedented time in neurotherapeutics. Medicines that address the causative disease biology underlying central nervous system
In a fireside chat at the American Society of Gene & Cell Therapy conference, CBER Director Peter Marks spoke with Takeda’s Kristin Van Goor about how the regulator is approaching the exploding gene therapy space.
While Teva missed Wall Street expectations in the first quarter of 2024, it reported Wednesday a 5% increase in Q1 revenues while reporting that the company’s schizophrenia candidate scored a late-stage trial victory
Regeneron Pharmaceuticals on Wednesday revealed that its investigational gene therapy DB-OTO restored hearing in two young children, according to an oral presentation at the American Society of Gene & Cell Therapy annual meeting.
Amylyx’s recent decision to withdraw its ALS drug Relyvrio from the market highlights an important business decision for companies: when to continue marketing or investigating a drug that has failed a pivotal or confirmatory study.
AstraZeneca announced that it will voluntarily pull Vaxzevria from the global market amid a sharp decline in demand and following the company’s recent admission that its vaccine is linked with a rare side effect.
As interest in psychedelic therapies ramp up, Lykos Therapeutics will go in front of the FDA’s Psychopharmacologic Drugs Advisory Committee on June 4 to present its investigational treatment for post-traumatic stress disorder.
Pfizer’s investigational Duchenne muscular dystrophy gene therapy fordadistrogene movaparvovec has been hit with another patient death, forcing the pharma to pause dosing in its Phase III study.
As Sarepta Therapeutics moves closer to full approval and an expanded label for its gene therapy, some experts push back on clinical efficacy and cost while others note the hope it provides patients with Duchenne muscular dystrophy.
The biotech touted its prime editing technology at ASGCT on Tuesday after receiving FDA clearance last week for a clinical study of a drug candidate based on the platform.