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.
Singapore’s Health Science Authority is rolling out new drug substance evidence requirements following consultations with stakeholders, and drugmakers will have one year to comply with the new regulations.
Bioxcel Therapeutics Inc. said a third-party audit of data from its Tranquility II phase III study came back clean, potentially positioning the firm to submit the statistically significant results in a supplemental NDA seeking approval of BXCL-501 in treating agitation associated with dementia in probable Alzheimer’s disease.
Four other companies want to replicate Biomarin Pharmaceuticals Inc.’s recent success as they seek approvals to treat the rare but most prevalent genetic form of dwarfism, achondroplasia. The U.S. FDA approved the sNDA for injectable Voxzogo (vosoritide), fibroblast growth factor receptor 3 (FGFR3) inhibitor from Biomarin on Oct. 20.
After nearly two years with an unconfirmed acting director at its helm, the U.S. NIH is one step closer to finally having Senate-confirmed leadership to steer the agency in a time pressed with political agendas, pandemic concerns and the potential for what could be life-altering innovations. The Senate Health, Education, Labor and Pensions (HELP) Committee voted 15-6 Oct. 25 to favorably report Monica Bertagnolli’s nomination to the full Senate for a confirmation vote as the next NIH director.
U.S. Sen. Bernie Sanders (I-Vt.) is calling for an investigation into the NIH’s proposed grant of an exclusive patent license for a late-stage cervical cancer drug to a small company linked to a former NIH researcher.
Biologics innovators typically take a lifecycle approach to developing new indications and formulations of their prescription drugs, especially when biosimilar competition is on the horizon.
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