Two therapies receive orphan drug status
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The FDA granted orphan drug designation to two therapies in development for hematologic malignancies.
Umbralisib — a dual inhibitor of the PI3 kinase-delta and CK1-epsilon — is being evaluated as treatment for several types of lymphoma as part of the phase 2b UNITY-NHL trial.
In one part of the study, researchers assessed the efficacy and safety of umbralisib for 118 patients with follicular lymphoma who received at least two prior lines of therapy, including an anti-CD20 monoclonal antibody and an alkylating agent. Overall response rate served as the primary endpoint.
The results met the company’s prespecified response target of ORR between 40% and 50%.
Another portion of UNITY-NHL designed to evaluate umbralisib for treatment of patients with marginal zone lymphoma also met its primary endpoint.
In January, TG Therapeutics initiated a rolling submission of a new drug application to the FDA for umbralisib as treatment for both lymphoma subtypes.
SEL120, 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.
“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,” Setareh Shamsili, MD, PhD, chief medical officer and executive vice president at Ryvu Therapeutics, said in a company-issued press release. “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.”
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.