The FDA has had a busy week. Two new cancer drugs have received approvals, one going to a pharmaceutical giant and the other to a company getting its first regulatory award. Meanwhile, three companies have received full response letters for various shortcomings. Here is an overview of recent FDA regulatory activity.
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—The FDA has approved a new cancer drug from Johnson & Johnson and its partner Legend Biotech, a CAR-T cell therapy for multiple myeloma. The treatment, called Carvykti, targets the BCMA protein on multiple myeloma cells. FDA approval of Carvykti makes it the second CAR-T therapy approved to treat blood cancers. Bristol Myers Squibb received approval for its CAR-T drug for multiple myeloma, Abecma, last year.
—CTI BioPharma has received FDA approval for pacritinib for the treatment of myelofibrosis in adults. Biotech Seattle will market its new drug, a twice-daily pill, under the name Vonjo.
Myelofibrosis is a cancer of the bone marrow that affects the production of red blood cells. The disease also causes severe thrombocytopenia, which is a dangerously low level of platelets. Vonjo is a small molecule designed to specifically block a Janus kinase (JAK) enzyme, the dysregulation of which has been implicated in myelofibrosis. The drug will compete with Jakafi, a JAK inhibitor from Incyte. Potential competition could also come from Sierra Oncology's mometinib, which recently published encouraging Phase 3 data that Biotech says will support regulatory filing plans.
- The FDA has rejected Amryt's application for approval of its treatment for epidermolysis bullosa, a rare inherited condition that causes fragile skin that is prone to blistering and cracking. The drug Amryt Oleogel-S10 is an oil-based gel intended to promote rapid healing of wounds. According to the company, the agency has requested additional information proving the drug's effectiveness. Amryt, based in Dublin, Ireland, said it plans to discuss with the FDA what kind of data is needed to address the agency's concerns.
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—Reata Pharmaceuticals has received a comprehensive response letter for bardoxolone, the company's investigational treatment for chronic kidney disease caused by the rare condition Alport syndrome. The drug Reata is a small molecule designed to activate a transcription factor that restores mitochondrial function, which in turn stops inflammation that damages the kidneys.
According to the Plano, Texas-based biotech, the FDA raised questions about safety and efficacy and asked the company to conduct another clinical trial to provide more data. Reata said he plans to work with the FDA to confirm the next steps for his drug.
—The FDA sent Gilead Sciences a comprehensive response letter for its HIV drug, lenacapavir, an antiviral drug designed to block HIV at multiple stages of its life cycle. According to the Foster City, Calif.-based company, the FDA cited manufacturing issues with the bottle. The company previously said the agency had questions about borosilicate glass vials and their compatibility with lenacapavir solution. Gilead said the FDA has not asked the company to conduct new clinical trials.