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The FDA is signaling change, but actual success depends on more than simply bringing in a new leader at the Center for Biologics Evaluation and Research; it requires accountability, transparency and consistent action.
FEATURED STORIES
With a greenlight for ibogaine to enter clinical testing and three unnamed products set to receive Commissioner’s National Priority Vouchers this week, it’s full speed ahead for psychedelics. But will sidestepping normal regulatory protocols actually be a net negative for the field?
With an IPO raise of $625 million, Kailera Therapeutics now holds the new record for the largest public market debut.
After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination of 20 years of hard work unraveling the mysteries of the blood-brain barrier.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
Doubling survival in pancreatic cancer, a long-fought rare disease approval, a massive IPO and ambitious biotech entrepreneurs have BioSpace Senior Editor Annalee Armstrong feeling upbeat about the biotech scene.
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Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a need for more rigorous evidence.
Pfizer has a lofty goal for the CDK4 inhibitor atirmociclib, the New York pharma’s answer to Ibrance’s loss of patent protection next year. In 2025, Ibrance led Pfizer’s oncology portfolio with $1.04 billion in sales.
The Phase 3 EMANATE study is a basket trial looking at the efficacy of Rhythm Pharmaceuticals’ injectable obesity drug across four types of obesities driven by specific genetic mutations.
Astellas Pharma and CytomX Therapeutics first partnered up in March 2020. For $80 million upfront, the Japanese pharma gained access to the biotech’s Probody platform to generate masked antibody therapies for cancer.
A Massachusetts judge called Kennedy’s efforts to reform the CDC’s vaccines advisory panel a “procedural failure,” adding that the new committee members do not “comport with governing law.”
In a highly competitive space, AI platforms must now prove themselves through proprietary data, focused pipelines and clinical readouts in competitive diseases. Promises of faster, cheaper drug discovery are not enough to entice strong investor engagement.
With Ascendis Pharma entering the achondroplasia space last month and BridgeBio on deck, BioMarin faces competition. Adding to the pressure, the company suffered a setback Monday when it halted two studies of Voxzogo in other growth-related conditions following multiple cases of hip injuries in other trials of the drug.
Structure Therapeutics’ oral obesity drug elicited a placebo-adjusted weight loss of 16.3% at 44 weeks in a Phase 2 trial. The biotech is planning to launch a late-stage program for the drug later this year.
Health Secretary Robert F. Kennedy Jr. has become increasingly unpopular among several government officials, largely as a result of his antivaccine rhetoric and actions. Other contentious issues reportedly include the approval of an abortion pill and other controversial FDA decisions.
Sana Biotechnology is looking to start clinical development for its type 1 diabetes therapy SC451 this year.