The FDA has had a busy week. Two new cancer drugs have been approved, one by the pharma giant and the other by the company to receive the first regulatory approval. Meanwhile, the three companies received full response letters for various deficiencies. Below is a review of recent FDA regulatory activity.
—FDA approved a Johnson & Johnson and Partner Legend Biotech’s Novel Cancer Drug, a CAR T-Cell Therapy for Multiple Myeloma. The treatment, called Carvykti, targets the BCMA protein on multiple myeloma cells. The FDA approval of Carvykti makes it the second CAR T therapy approved for the treatment of blood cancers. Bristol-Myers Squibb’s multiple myeloma CAR T drug Abecma won regulatory nod last year.
—CTI BioPharma awarded FDA Approval of pacitinib as a treatment for myelofibrosis in adults. The Seattle biotech will market its new drug under the name “Vonjo,” a twice-daily pill.
Myelofibrosis is a cancer of the bone marrow that affects red blood cell production. The disease can also cause severe thrombocytopenia, a dangerously low level of platelets. Vonjo is a small molecule designed to specifically block Janus kinase (JAK), the dysregulation of which is associated with myelofibrosis. The drug will compete with Jakafi, a JAK inhibitor from Incyte.Potential competition could also come from Sierra Oncology’s momelotinib, recently released encouraging Phase 3 data The biotech company said it would support plans to submit regulatory filings.
—U.S. Food and Drug Administration reject Amryt files for approval to treat epidermolysis bullosa, a rare genetic disorder that causes fragile skin to blister and tear easily. Amryt’s drug Oleogel-S10 is an oil-based gel designed to promote rapid wound healing. The agency requested more information showing the drug’s efficacy, according to the company. Dublin, Ireland-based Amryt said it plans to discuss with the FDA the nature of the data needed to address the agency’s concerns.
—Reata Pharmaceuticals received full response letter on bardoxolonethe company’s experimental treatment for chronic kidney disease Caused by the rare disease Alport syndrome. The Reata drug is a small molecule designed to activate transcription factors that restore mitochondrial function, thereby stopping inflammation that damages the kidneys.
According to the Plano, Texas-based biotech, the FDA has questioned safety and efficacy and asked the company to conduct another clinical trial to provide more data. Reata said it plans to work with the FDA to confirm the next steps for its drug.
—FDA issued a complete reply letter Gilead Sciences’ HIV drug lenacapavir, an antiviral drug designed to block HIV at multiple stages of its life cycle. The FDA cited manufacturing issues with the vials, according to the Foster City, California-based company. The company had previously announced that the agency had questions about the vial made of borosilicate glass and its compatibility with the lenakapavir solution. Gilead said the FDA has not asked the company to conduct any new clinical trials.
—FDA placed a clinical hold In Phase 3 testing of Finch Therapeutics’ experimental treatment for recurrent Clostridium difficile infectionsAccording to the Somerville, Massachusetts-based biotechnology company, regulators are requesting more information on SARS-CoV-2 donor screening protocols.
Finch’s therapy CP101 is made from feces from healthy donors. Microbiome therapy aims to restore a patient’s gut bacteria to a healthy state. At the start of the pandemic, the FDA issued an alert about the potential risk of transmission of Covid-19 from donor-derived microbiome therapies. According to Finch, the FDA sent letters in January and February requesting more information about the company’s SARS-CoV-2 screening program. In particular, FDA wants more details on how samples are shipped to suppliers who perform screening and how inconclusive test results are handled. Finch said it will quickly provide the information needed and resolve the clinical hold.
Food and Drug Administration photo
