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While tovecimig met the main goal of progression-free survival in a Phase 2/3 trial, it did not improve overall survival.
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As cell and gene therapy leaders gathered in Maryland to discuss accelerating clinical trials in children, one “cutting edge” session focused on the need to expedite more bespoke gene editing treatments like the one that saved young KJ Muldoon.
Neal and Azbee awards have validated our approach to reporting on the industry at a time of unprecedented shifts at the FDA and other federal agencies.
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.
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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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While Johnson & Johnson retains the top revenue rank across the major pharma companies, Eli Lilly last year established itself as the clear leader in the obesity market, in the process capturing investors’ attention and enthusiasm.
The major pharmas are loaded up with trillions in firepower—but are sticking to mid-cap deals. One expert says it might be time to think outside the box and shake up the industry with some consolidation.
In its complete response letter, the FDA said Aldeyra had failed to demonstrate reproxalap’s efficacy in adequate and well-controlled studies. The FDA previously turned the candidate away in November 2023 and April 2025.
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.