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Sanofi will take venglustat to regulators for Gaucher disease but an application for Fabry disease is less clear after the failure of a Phase III trial.
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With Lykos’ regulatory failure now squarely in the rearview mirror, Compass Pathways and Definium are leading what one analyst suspects will be “a very big year for psychedelics.”
The Senate failed to pass a massive spending bill on Thursday—which includes the rare pediatric PRV program but also funding for the Immigration and Customs Enforcement’s large-scale crackdown in Minnesota and other states.
With the biopharma industry performing better of late, analysts, executives and other industry watchers are “cautiously optimistic”—a term heard all over the streets of San Francisco at the J.P. Morgan Healthcare Conference earlier this month.
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Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
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The bladder cancer-focused company’s larger-than-expected Nasdaq debut on Thursday opens this year’s batch of planned initial public offerings, which include at least half a dozen biotechs.
More than 3,000 people in the U.S. suffered from side effects of Ozempic in 2023, according to exclusive reporting on Wednesday from Reuters citing data from America’s Poison Centers.
After 26 weeks of follow-up, five of six children treated with an experimental gene therapy developed in China showed strong improvements in speech perception and could hold normal conversations.
The FDA’s Peter Marks and Nicole Verdun in the NEJM Wednesday disclosed that more than 27,000 CAR-T doses have been administered in the U.S. as of the end of 2023, of which there are 22 cases of T-cell cancers.
Most respondents expressed concerns about the economic environment, as competition for jobs jumped more than 130%.
In the biopharma industry, engineers are pivotal drivers of innovation, helping to shape the landscape of healthcare discoveries.
While the FDA considers a T cell malignancy risk to be applicable to all commercial CAR-T therapies, Gilead Sciences’ Tecartus has a revised warning that potential adverse events “may” occur.
The regulator’s Complete Response Letter flagged issues with the new formulation of tesamorelin’s chemistry, manufacturing and controls and sought more information about its immunogenicity risk.
While decentralized trials are expediting innovation and promoting diversity, challenges remain, including complex stakeholder ecosystems, skills gaps and regulation.
The company said it’s preparing to file a New Drug Application with the FDA on the strength of late-stage study data for its investigational RNA-targeted donidalorsen in patients with hereditary angioedema.