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The FDA has renewed calls for Amgen’s Tavneos to be pulled from the market, saying it has discovered new evidence that study personnel doctored the results of the drug’s pivotal study in order to make it look effective.
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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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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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Ceralasertib is part of AstraZeneca’s ambitious plan to hit $80 billion in revenue by 2030.
Jacobio discovered JAB-23E73, which is designed to treat several KRAS mutation subtypes, and is testing the therapy in multiple Phase I trials.
Stifel analysts said the label for cardiac myosin inhibitor Myqorzo is in line with their expectations and is differentiated compared with BMS’ Camzyos.
Pfizer and Metsera, Sarepta and uniQure made the list with dramatic tales. The other two spots went to the regulatory challenges facing biopharma under the new administration, especially in the vaccines sector.
The second half finished strong after two tumultuous years. What will 2026 bring for the biotech sector?
Policy initiatives have come fast and furious at the FDA this year. While guidances on rare diseases and vaccines have consumed most of the ink, policy shifts aimed at improving FDA efficiencies and reshoring U.S. manufacturing also got some attention. Here, BioSpace rounds up more than a dozen initiatives relevant to the biopharma industry.
A report from analysts at Jefferies suggested that new screenings for metachromatic leukodystrophy and Duchenne muscular dystrophy could bump sales of the gene therapy Libmeldy by more than $100 million.
BioMarin Pharmaceutical has faced a rocky road, promising and then backing off revenue targets and cutting assets that have underperformed. But Amicus’ rare disease portfolio is already bringing in $600 million annually.
After 27 years in business, Cytokinetics hopes to pit its own cardiac myosin inhibitor against one it initially developed—now owned by Bristol Myers Squibb—in a market worth billions. Aficamten has a PDUFA date of Dec. 26.
Insmed pointed to a strong placebo response as the reason for the trial’s failure.