Here’s the latest in drug approval news, including new approvals, notable trial readouts and other development updates.
The FDA approved Imaavy (diaminopyrimidine-acetyl sulfinic acid) to treat generalized myasthenia gravis, offering hope for those who have not found relief with other treatments. This once-daily pill reduces symptoms and autoantibody levels and may also slow kidney damage.
In Europe, a revolutionary gene therapy gained fast-track approval. Aucatzyl, a CAR T-cell immunotherapy, is the first treatment for an aggressive blood cancer called B-cell acute lymphoblastic leukemia to be approved under a centralized review procedure at the European Medicines Agency. The therapy is expected to cost $6 million per patient, according to Evaluate, which estimates 2030 sales at $3.6 billion.
TIDES and other biologics have dominated the new drugs approved this month, but there were also some new small-molecule drug approvals, including nedosiran (RivflozaTM), a 58-nucleotide oligonucleotide that treats a rare genetic disease, tenosynovial giant cell tumor of the joints (TGCT). This is one of nine oligos and five peptides to receive FDA approval this year, including Vertex Pharmaceuticals’ Vanza triple combination for cystic fibrosis (Alyftrek), which is predicted to reach $8.3 billion in annual sales.
FDA Commissioner Marty Makary has vowed to speed up reviews of promising medicines, and a new program will allow companies that have been granted national priority designation to skip the normal clinical trials required for most drugs and get to market in six months instead of 10. This program is similar to an accelerated approval program the FDA launched last summer, which lets it approve life-saving treatments based on safety data and images showing tumor shrinkage without waiting for randomized clinical trial results. But some scientists argue that accelerated approval programs haven’t resulted in better outcomes for patients and could lead to drugs that end up being unsafe or ineffective.
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