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.
Advocates of expanded use of telehealth in the U.S. may believe they have an unfairly high evidentiary bar to meet to bring payers on board, but that evidentiary requirement just received support from the National Institutes of Health (NIH).
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Alivedx, Neurotrigger.
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.
Regulatory snapshots, including global submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Cordance, Exatech.
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.
The FDA has accepted Mustang Bio Inc.’s IND application of MB-109 for the treatment of recurrent glioblastoma (GBM) and high-grade astrocytoma. MB-109 is a treatment regimen combining MB-101 (City of Hope-developed IL13Rα2‐targeted CAR T-cell therapy) and MB-108 (Nationwide Children’s Hospital-developed HSV-1 oncolytic virus).
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