March 05, 2020: TG Therapeutics announced that the U.S.FDA granted orphan drug designation to umbralisib, the Company’s investigational dual inhibitor of PI3K-delta and CK1-epsilon, for the treatment of patients with follicular lymphoma (FL).
“In the UNITY-NHL Phase 2b registration controlled clinical trial, Umbralisib is tested across several types of lymphoma.
The UNITY-NHL trial FL cohort is designed to determine the safety and efficacy of umbralisib in FL patients who have undergone at least two prior therapy lines including a monoclonal antibody anti-CD20 and an alkylating agent.
The Company announced in October 2019 that the FL cohort reached the primary endpoint of the overall response rate (ORR), and the Company introduced a rolling submission of a New Drug Submission (NDA) to the US in January.
Administration of Food and Drugs (FDA) for umbralisib as a treatment for patients with previously diagnosed marginal zone lymphoma (MZL) and FL. For the treatment of patients with all three forms of MZL, the FDA previously granted umbralisib orphan drug designation: nodal, extranodal, and splenic MZL.”
The U.S. grants orphan drug designation. “FDA on drugs and biologics identified as those intended for safe and effective treatment, diagnosis or prevention of rare diseases/disorders affecting fewer than 200,000 people in the United States. The classification of orphan drugs includes other benefits that can include tax deductions against the expense of clinical trials and exemptions for prescription drug users. When, eventually, a product with an orphan drug label earns the first FDA approval for the disease for which it has that designation, the product is entitled to exclusivity for orphan drugs.”
“Follicular lymphoma (FL) is usually a slow-growing or indolent type of non-Hodgkin lymphoma (NHL) which results from B-lymphocytes, making it a lymphoma of the B-cells. Follicular lymphoma is typically not curable, and it is a chronic condition. Patients with this form of lymphoma will live with it for many years. With an estimated occurrence of about 15,000 newly diagnosed patients in the United States, FL is the most common indolent lymphoma, accounting for about 20 per cent of all NHL cases.”
https://fda.einnews.com/pr_news/511287565/tg-therapeutics-receives-orphan-drug-designation-for-umbralisib-from-the-u-s-food-and-drug-administration-for-the-treatment-of-follicular-lymphoma
