FDA grants SEL120 orphan drug designation for acute myeloid leukemia
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The FDA granted orphan drug designation to SEL120 for the treatment of acute myeloid leukemia, according to the agent’s manufacturer.
SEL120 (Ryvu Therapeutics), an investigational oral CDK8 kinase inhibitor, is being tested for safety and efficacy in a phase 1b clinical trial of patients with relapsed or refractory AML or high-risk myelodysplastic syndrome.
“The FDA’s granting orphan drug designation to SEL120 is a significant encouragement for advancement of our clinical strategic plan addressing the unmet medical needs in the area of AML treatment, a disease where patients still face poor prognosis,” Setareh Shamsili, MD, PhD, chief medical officer and executive vice president at Ryvu Therapeutics, said in a company-issued press release.
“SEL120 has shown strong proof of concept in preclinical studies and has received strategic support from the Leukemia & Lymphoma Society through its Therapy Acceleration Program,” Shamsili added. “SEL120 may have the potential to offer an important therapeutic benefit in AML, and in particular to those patients with refractory or relapsed AML with the poorest prognosis, worldwide.”
The Therapy Acceleration Program is separate from the nonprofit’s normal grant process and “identifies and funds innovative projects related to therapies that have the potential to change the standard of care for patients with blood cancer, especially in areas of high unmet medical need,” according to its website. The program provides funding to help both investigators and commercial biotech companies develop clinical proof-of-concept data that can lead to new treatments for leukemia, lymphoma and myeloma.
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.