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.
Shares in Sierra Oncology Inc. (NASDAQ:SRRA) surged 38.5% to $54.75 on April 13 as Glaxosmithkline plc made a $55-per-share offer for the company, which values it at $1.9 billion. The offer represents a premium of about 39% on Sierra’s April 12 closing share price of $39.52 and a premium of about 63% over its volume-weighted average share price over the last 30 trading days.
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
Partners Sanofi SA and Swedish Orphan Biovitrum AB (Sobi) said regulatory submissions are expected this year for once-weekly factor VIII therapy efanesoctocog alfa in hemophilia A following top-line success in a pivotal phase III study, which showed a clinically meaningful prevention of bleeds in people with severe disease receiving prophylaxis over 52 weeks. The drug, also known as BIVV-001, has fast track and orphan designations in the U.S., and the companies are banking on its extended half-life to go up against blockbuster bispecific antibody Hemlibra (emicizumab) from Roche Holding AG as well as a potential gene therapy from Biomarin Pharmaceutical Inc.
RSS
