The FDA had a busy week. Two new cancer drugs secured approvals, one going to a pharmaceutical giant and the other to a company getting its first regulatory nod. Meanwhile, three companies were handed complete response letters for various deficiencies. Here’s a look back at the FDA’s recent regulatory activity.
—The FDA approved a new cancer drug from Johnson & Johnson and partner Legend Biotech, a CAR T-cell therapy for multiple myeloma. The treatment, named Carvykti, targets the BCMA protein on multiple myeloma cells. FDA approval of Carvykti makes it the second CAR T-therapy approved for treating the blood cancer. Bristol Myers Squibb won the regulatory nod for its multiple myeloma CAR T-drug, Abecma, last year.
—CTI BioPharma won FDA approval for pacritinib as a treatment for myelofibrosis in adults. The Seattle biotech 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 leads to severe thrombocytopenia, which is a dangerously low level of platelets. Vonjo is small molecule designed to specifically block a Janus kinase enzyme (JAK) whose dysregulation is associated with myelofibrosis. The drug will compete against Jakafi, a JAK inhibitor from Incyte. Potential competition could also come from Sierra Oncology’s momelotinib, which recently posted encouraging Phase 3 data that the biotech said will support plans for a regulatory submission.
—The FDA turned down Amryt’s application seeking approval of its treatment for epidermolysis bullosa, a rare, inherited disorder that leads to fragile skin prone to blisters and tearing. Amryt drug Oleogel-S10 is an oil-based gel intended to promote rapid wound healing. According to the company, the agency asked for additional information showing the drug’s efficacy. Dublin, Ireland-based Amryt said it plans to discuss with the FDA the nature of the data needed to address the agency’s concerns.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
—Reata Pharmaceuticals received a complete response letter for bardoxolone, the company’s experimental treatment for the chronic kidney disease caused by the rare disorder Alport syndrome. The Reata drug is a small molecule designed to activate a transcription factor that restores mitochondrial function, which in turn stops the inflammation that damages the kidneys.
According to the Plano, Texas-based biotech, the FDA raised questions about both safety and efficacy, and asked the company to conduct another clinical trial to provide more data. Reata said that it plans to work with the FDA to confirm the next steps for its drug.
—The FDA issued a complete response letter to Gilead Sciences for its HIV drug lenacapavir, an antiviral designed to block HIV at multiple stages of its life cycle. According to the Foster City, California-based company, the FDA cited manufacturing issues with the vial. The company had previously announced that the agency had questions about vials made of borosilicate glass and their compatibility with lenacapavir solution. Gilead said that the FDA did not ask the company to conduct any new clinical trials.
—The FDA placed a clinical hold on a Phase 3 test of Finch Therapeutics’ experimental treatment for recurrent C. difficile infection. According to the Somerville, Massachusetts-based biotech, the regulator is asking for more information about SARS-CoV-2 donor screening protocols.
Finch’s therapy, CP101, is made from stool sourced from healthy donors. The microbiome treatment is intended to restore a patient’s gut bacteria to a healthy state. At the beginning of the pandemic, the FDA issued an alert about the potential risk of Covid-19 transmission from donor-derived microbiome therapies. According to Finch, the FDA sent letters in January and February asking for more information about the company’s SARS-CoV-2 screening protocols. In particular, the FDA wants more detail on how samples are shipped to the vendor performing the screening and how inconclusive test results will be handled. Finch said it will work quickly to provide the requested information and resolve the clinical hold.
Photo by FDA
