All Clarivate websites use cookies to improve your online experience. They were placed on your computer when you launched this website. You can change your cookie settings through your browser.
Alexion Pharmaceuticals Inc. has demonstrated the commercial potential for rare disease drugs with its complement inhibitor Soliris (eculizumab) and long-lasting follow-up Ultomiris (ravulizumab) driving blockbuster sales. A host of other companies are hoping to compete with Ultomiris, which is U.S. FDA-approved for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Sponsors developing PI3K inhibitors to treat hematologic malignancies in the U.S. may have to up their game if they want to get FDA approval. The Oncologic Drugs Advisory Committee voted 16-0, with one abstention, that future approvals of the drugs should be supported by randomized data. Given the FDA’s briefing document ahead of the April 21 meeting and its presentations to the committee, the agency is likely to follow that recommendation.
It’s been one thing after another for rusfertide from Protagonist Therapeutics Inc. for the past year. Now the U.S. FDA has told the company it wants to rescind the breakthrough designation, a relatively rare occurrence, based on observed malignancies in a follow-up to September’s clinical hold.
