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A federal appeals court has rejected Jazz Pharmaceuticals’ attempt to overturn a lower court ruling invalidating a patent claim covering the company’s Risk Evaluation and Mitigation Strategies (REMS) distribution system for its blockbuster narcolepsy therapy Xyrem (oxybate). Read More
The FDA is bringing in ethicists to help the agency better frame issues around gene editing as well as data generation using patients with rare diseases that have no cure, said FDA Commissioner Robert Califf, speaking during the agency’s Rare Diseases Day 2023 virtual public meeting on Monday. Read More
Cosmoceutical Research Center was hit with a five-observation Form 483 following an inspection of its Panorama City, Calif., facility for lack of written quality control procedures, failing to test incoming components and several other lapses. Read More
Attorneys general from a dozen states have filed a lawsuit against the FDA over its Risk Evaluation and Mitigation Strategies (REMS) for the abortion pill, mifepristone, arguing that the restrictions are not necessary and create burdens for patients. Read More
A single infusion of BioMarin’s Roctavian (valoctocogene roxaparvovec) reduced bleeding rates by 84 percent for two years in patients with hemophilia A, according to a study published in The New England Journal of Medicine (NEJM). Read More
The FDA will soon announce a new pilot program to help developers of gene therapies for rare diseases get their treatments approved faster, said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), at the agency’s Rare Diseases Day 2023 virtual public meeting on Monday. Read More