As a follow-up to the Biden administration’s executive order for artificial intelligence (AI), the U.S. Office of Management and Budget (OMB) has promulgated a memorandum directing federal government agency use of AI.
Had it been asked to, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee would have voted Oct. 31 to recommend approval of Vertex Pharmaceutical Inc. and Crispr Therapeutics AG’s exagamglogene autotemcel, or exa-cel, as a one-time transformative treatment for severe sickle cell disease in individuals 12 and older.
The Biden administration has issued an executive order (EO) for artificial intelligence (AI), which addresses not only national security considerations, but public health considerations as well. One of the features of the EO is that any company developing AI for public health and safety must notify the federal government when training a foundation AI algorithm, which suggests that AI that is regulated by the U.S. FDA will now be subject to additional government scrutiny in the premarket phase.
Patients living with aggressive nasopharyngeal carcinoma (NPC) now have a new treatment option with the U.S. FDA approval of Junshi Biosciences Co. Ltd./Coherus Biosciences Inc.’s PD-1 inhibitor antibody Loqtorzi (toripalimab), which will likely become the new standard of care for NPC.
Most of the patients and advocacy groups speaking at the first of 10 public listening sessions questioned the Biden administration’s talking points that U.S. Medicare’s prescription drug price negotiation will be good for beneficiaries because it will improve access to costly drugs by lowering prices.
It’s just a discussion next week of Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG’s sickle cell disease (SCD) therapy, with no vote expected from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee. The FDA isn’t questioning the drug’s efficacy as much as it is concerned about the effects of off-target editing.
Less than two weeks after getting a thumbs-up from the EMA’s Committee for Medicinal Products for Human Use, Santhera Pharmaceutical AG’s vamorolone secured U.S. FDA approval for use in patients, 2 and older, with Duchenne muscular dystrophy (DMD). A first-in-class drug, vamorolone, branded Agamree, is expected to offer a safer alternative to the steroid therapy, which the company has said will remain a foundational treatment of DMD, even with the introduction of gene therapies.
After a delay caused by the April complete response letter from the U.S. FDA for Eli Lilly and Co.’s Omvoh (mirikizumab-mrkz), regulators cleared the drug for moderately to severely active ulcerative colitis (UC) in adults. Given by infusion (300 mg/15 mL)/injection (100 mg/mL), Omvoh stands as the first and only interleukin (IL)-23p19 antagonist for the treatment of moderately to severely active UC in adults – as well as the pharma giant’s first drug approval in the space.
In a show of bipartisan solidarity, members of the U.S. Senate Special Committee on Aging voiced their support Oct. 26 for a new regulatory pathway to quicken access to new drugs for rare diseases that have no approved treatments.
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