Regulatory
Developers of psychedelics-based therapies say the industry is poised to explode, with several reporting strong clinical trial results and the FDA granting breakthrough status for two hallucinogenic drugs.
The FDA’s new guidance on decentralized clinical trials clarifies how investigators and trial sponsors can use digital health technologies and involve primary healthcare practitioners.
The FDA’s agenda includes an advisory committee meeting for Sarepta’s delandistrogene moxeparvovec, which, if approved, would become the first gene therapy for Duchenne muscular dystrophy.
For people with, or at risk for, SOD1-ALS, the FDA’s approval of Qalsody is an important step toward advancing gene-specific research for this uniformly fatal disease.
Merck and AstraZeneca are seeking broad approval for Lynparza in mCRPC regardless of BRCA status.
The regulatory filing puts the company alongside Vertex and CRISPR, which also await FDA approval for their SCD gene therapy.
Tuesday, the FDA approved Qalsody—formerly tofersen—on a conditional basis to treat SOD1-ALS.
The partial clinical hold follows Foghorn’s voluntary pause of the study and is due to one patient developing irregular heartbeat following treatment with FHD-609.
Eplontersen halted ATTRv-PN disease progression and improved quality of life through 66 weeks. The drug has a PDUFA date of Dec. 22, 2023.
The FDA’s Center for Biologics Evaluation and Research is aiming to recreate the success achieved with the rapid development of COVID-19 vaccines under a program of the same name.
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