FDA
After a year of controversial regulatory reversals, the FDA has accepted uniQure’s application for ifezuntirgene inilparvovec for priority review, with a May 1 action date. If approved, the therapy—also called AMT-130—would be the first disease-modifying treatment for Huntington’s disease.
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Disc Medicine’s bitopertin—rejected by the FDA in February—significantly reduced levels of a key disease biomarker and significantly increased the time patients with erythropoietic protoporphyria were able to spend in the sun and artificial light.
Leveraging recently proposed regulatory flexibilities, the gene-editing sector is working to cut R&D costs and unlock indications that were previously economically unviable, according to a new playbook published by the Alliance for Regenerative Medicine.
The FDA has taken important steps through Operation TrialBlazer and stronger scrutiny of foreign clinical data, but more must be done to make the United States more competitive in attracting clinical trials as China’s capabilities rise.
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Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
The approval of Hibsago has created a new market for a handful of biopharmas, including Gilead, Arrowhead, and Vir, to fill in.
The recurrence of violations at multiple sites led the FDA to conclude that Baxter management must improve its oversight and control of manufacturing.
Hibsago’s approval—a functional cure for certain patients with a hepatitis B infection—further validates Ionis Pharmaceuticals’ antisense platform, which has already generated two other new drug approvals this year.
Inconsistent reporting at a Pennsylvania site affected microbiological analyses of components and drug products that analytical testing service provider Eurofins ran for customers.
Following several recent initiatives, the FDA’s newest rare disease framework sets out to standardize the agency’s approach to scientific evidence and bring rare disease therapies to patients faster.
After one patient death prompted an FDA hold this spring, Newron Pharmaceuticals has now shared that a total of four fatalities have been recorded among participants receiving its schizophrenia candidate. A fifth death was recorded among those taking the placebo.
Despite the regulatory delay, Novo is still expecting a 2027 launch for its hemophilia A drug denecimig.
Patients had adverse events after receiving a Fresenius Kabi medicine that experienced manufacturing deviations.
Amid rising industry concerns about U.S. competitiveness with China, the Trump administration is rolling out four ARPA-H initiatives to modernize clinical trial design, site activation, consent and patient data collection.