FDA grants orphan drug designation to umbralisib for follicular lymphoma
The FDA granted orphan drug designation to umbralisib for the treatment of follicular lymphoma, according to the agent’s manufacturer.
Umbralisib (TG Therapeutics) — 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 as treatment 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.
“The receipt of orphan drug designation for umbralisib to treat patients with follicular lymphoma is another important milestone in the development and anticipated commercialization of umbralisib in marginal zone lymphoma and follicular lymphoma,” Michael S. Weiss, executive chairman and CEO of TG Therapeutics, said in a company-issued press release. “We are excited by the progress so far and look forward to completion of this submission targeted in the first half of this year.”
The FDA previously granted orphan drug designation to umbralisib for all three types of marginal zone lymphoma: nodal, extranodal and splenic.
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.