FDA
FDA OKs Takeda, Protagonist drug for rare blood cancer
The U.S. FDA has approved Mimrylo (rusfertide), a first-in-class drug developed in collaboration between Takeda Pharmaceutical and Protagonist Therapeutics, for the treatment of erythrocytosis in adult patients with polycythemia vera. The drug mimics hepcidin activity to reduce red blood cell production, and a Phase III trial showed that 77% of patients did not require phlebotomy. Mimrylo is priced at $4,200 per vial, and its label does not restrict prior treatments; analysts expect peak annual sales to reach $2 billion.
FDA green lights Priovant drug for rare skin disease
The U.S. FDA on Thursday approved the first oral drug brepocitinib (brand name Lisraya) for the treatment of dermatomyositis, a rare and debilitating skin disease. The drug was initially discovered by Pfizer and licensed to Priovant Therapeutics, a subsidiary of Roivant Sciences. The list price for a 30-day course is $35,000.
Regeneron to challenge Ipsen as FDA clears bone disease drug
The U.S. Food and Drug Administration (FDA) has approved Regeneron Pharmaceuticals' Pasatru (garetosmab) for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultra-rare degenerative bone disease. This clearance positions Pasatru to directly challenge Ipsen's Sohonos, which has seen sluggish sales since its 2023 approval. Regeneron's drug demonstrated a 90% or greater reduction in new heterotopic bone formation over 12 months in a pivotal Phase 3 trial, despite a development history marked by a clinical hold following participant deaths. The launch comes at a critical time for Regeneron, which faces biosimilar pressure on its top-selling eye drug Eylea and recent clinical setbacks in oncology.
Trump nominates policy aide Overton to lead FDA
President Donald Trump on Wednesday nominated Heidi Overton, a top White House policy aide and physician, to serve as FDA commissioner. The announcement, made by HHS Secretary Robert F. Kennedy Jr., comes amid ongoing turmoil at the agency. Overton, who lacks drug regulatory experience, would face Senate confirmation and questions about her policy direction.
Bristol Myers wins ‘milestone’ FDA approval of myeloma drug acquired from Celgene
Bristol Myers Squibb received FDA accelerated approval for Zenbexus, a CELMoD therapy acquired via Celgene, for second-line multiple myeloma. The approval is seen as a milestone, with analysts noting the portfolio's underappreciated value. Zenbexus, priced at $28,000 monthly, showed superior MRD-negative rates in a Phase 3 trial. A second CELMoD, mezigdomide, awaits FDA decision by May 13.
Trump signs order seeking to upend childhood vaccine schedule
U.S. President Trump signed an executive order on Monday named the "Golden Standard Childhood Vaccine Recommendations," attempting to make major changes to the U.S. childhood immunization program that has been in place for decades. The order plans to reduce the number of universally recommended vaccines from 17 to 11 and requires the measles-mumps-rubella (MMR) vaccine to be split into three separate injections. This move has been criticized by professional organizations such as the American College of Physicians for bypassing existing scientific review procedures. Additionally, the legal effect and scope of implementation of the executive order remain unclear, as vaccine mandate policies are primarily determined by individual states.
Replimune rebounds to win FDA approval of melanoma drug
The U.S. FDA approved Replimune's advanced melanoma therapy Tudriqev (formerly RP1) on Thursday, after two prior rejections. This is an accelerated approval requiring a subsequent Phase III trial to confirm efficacy. Data showed tumor responses in about a quarter of patients, with a median duration exceeding 14 months.
Takeda approval ushers in new wave of sleep drugs
The U.S. Food and Drug Administration (FDA) approved Takeda Pharmaceutical's Orzeyful (generic name: oveporexton) for the treatment of type 1 narcolepsy, making it the first orexin receptor agonist targeting the root cause of the disease. Analysts predict its peak annual sales could reach $2 billion. Companies such as Eisai, Eli Lilly, and Alkermes are also racing to develop similar drugs, indicating significant market potential.
Erica Schwartz confirmed as next CDC director
The U.S. Senate confirmed Erica Schwartz as CDC director on Wednesday, ending the agency's nearly year-long period without a permanent leader. During her confirmation hearing, Schwartz pledged to 'never betray science,' but some Democratic lawmakers expressed doubts about her ability to withstand political interference. The vote largely followed party lines, with the only Democrat voting in favor being Senator Tim Kaine of Virginia.
Replimune melanoma drug wins support of FDA panel
The U.S. FDA advisory committee voted 10 to 3 on Thursday to support Replimune's experimental melanoma treatment drug RP1, finding the efficacy results of its key study to be "assessable and clinically meaningful." Despite earlier concerns from FDA scientists about insufficient efficacy evidence, the majority of committee members believed that the tumor response signals observed in the refractory patient population warranted support. The FDA is expected to make a final approval decision by August 2.