Kadmon Holdings Inc (NASDAQ: KDMN) recently announced that the FDA (Food and Drug Administration) finally approved their REZUROCK (belumosudil) 200 mg once a day in treating adult and pediatric patients above 12 years old with chronic graft versus host disease (cGVHD). The drug is to be used after two failed attempts of other lines of systematic therapy.
The FD A gave Breakthrough Therapy designation and priority review for the drug and reviewed the NDA (New Drug Application) under the Real-Time Oncology Review pilot program. As a result, its approval came six weeks ahead of the PDUFA (Prescription Drug User Fee Act) goal date of August 30, 2021.
Breakthrough in cGVHD treatment
The drug is the first and only so far, FDA-approved molecule inhibitor of ROCK2, a signaling pathway that modulates fibrotic processes and inflammatory responses. Thus, it offers a whole new treatment paradigm for countless cGVHD patients, including a good number who have difficult to treat manifestations such as fibrosis. Corey Cutler, Associate Professor at Harvard, remarks that the drug has shown robust and durable respons4es in all the tests and can be approved as safe and well-tolerated by cGVHD patients. This allows them to stay on the course of therapy and achieve substantial benefits from the treatment.
The FDA approval of the drug is based on safety and efficacy findings from a randomized multi-center open-label trial with cGVHD patients who had undergone up to five prior lines of systematic therapy.
The trial involved 65 patients who took REZUROCK 200mg orally every day. The median time from diagnosis was 25.3 months, and almost half the patients had four or more organs affected. The patients had tried a median of 3 prior lines of systematic therapy, and close to 80% were refractory to their last treatment.
Great overall response
The drug’s overall response rate (ORR) was 75% by the first day of cycle 7 of treatment, with 69% achieving full response and 6% getting a partial response. The median first time for response was 1.8 months, and 62% of responders did not require systematic therapy for another year.