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Nurix Announces U.S. FDA Orphan Drug Designation Granted to Bexobrutideg (NX-5948) for the Treatment of Waldenström Macroglobulinemia
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström Macroglobulinemia First-in-class Bruton’s
About this update from Nurix Therapeutics, Inc.
[{"type":"text","content":"Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström Macroglobulinemia First-in-class Bruton’s tyrosine kinase (BTK) degrader NX-5948 assigned nonproprietary name “bexobrutideg” in newly named degrader class of drugs SAN FRANCISCO, March 17, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to bexobrutideg (NX-5948) for the treatment of Waldenström macroglobulinemia (WM). Bexobrutideg is an orally bioavailable, brain penetrant degrader of BTK which is being evaluated in an ongoing Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies. The FDA's Orphan Drug Designation program provides orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. This designation provides certain benefits, including tax credits for qualified clinical testing, waiver or partial payment of FDA application fees and seven years of market exclusivity, if approved. “The FDA’s Orphan Drug Designation for bexobrutideg, also known as NX-5948, represents an important milestone in our regulatory strategy and underscores the significant unmet medical need for improved treatments for Waldenström macroglobulinemia,” said Arthur T. Sands, M.D., Ph.D., president and chief executive officer of Nurix. “Granting of the designation highlights bexobrutideg’s potential to provide patients with WM a promising new therapeutic option. We are also pleased to announce that our investigational therapy bexobrutideg has been assigned a nonproprietary name reflecting its novel mechanism of action, designated with the unique suffix “deg” for degrader.” In collaboration with the national naming authority, United States Adopted Name (USAN) Council, Nurix’s lead BTK degrader, NX-5948, was assigned the nonproprietary name “bexobrutideg.” The U.S. and international drug naming convention is designed to select a single name of worldwide acceptability for each active substance that is intended to be marketed as a pharmaceutical. Most nota...