Regulatory
If approved by the FDA, the MDMA-assisted treatment for individuals with post-traumatic stress disorder would be the first U.S. psychedelic-assisted therapy.
The agency is investigating reports of secondary blood cancers in patients who have received certain CAR T cell therapies, but experts say the risk is low and is outweighed by the terminal nature of some cancers.
The year may be coming to a close, but there’s no holiday break for the FDA, which will release five regulatory verdicts over the next two weeks.
Medicines on the list can still be prescribed but will be under tight monitoring by a regulatory network, which can implement measures to prevent shortages and ensure supply chain continuity.
Ahead of an expected surge in regulatory filings, the FDA is establishing the Genetic Metabolic Diseases Advisory Committee to provide advice on treatments for these complex and challenging conditions.
The companies have received all required regulatory approvals to complete the deal, the largest for the sector in the past three years and the biggest for the hot antibody-drug conjugate market.
Following the regulator’s administrative complaint and threat of a lawsuit in federal court, Sanofi has decided to terminate its licensing deal with Maze Therapeutics to avoid a long litigation process.
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
Along with CRISPR/Cas9-based Casgevy—developed by Vertex Pharmaceuticals and CRISPR Therapeutics—the regulator on Friday approved bluebird bio’s Lyfgenia, a second gene therapy for sickle cell disease.
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
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