Nurix Therapeutics Receives Orphan Drug Designation for Bexobrutideg in Lymphoplasmacytic Lymphoma

Nurix Therapeutics has received Orphan Drug Designation from the European Medicines Agency for bexobrutideg (NX-5948) to treat lymphoplasmacytic lymphoma, a rare form of cancer. Bexobrutideg is an orally bioavailable, brain penetrant degrader of Bruton’s tyrosine kinase (BTK) being evaluated in a Phase 1a/b clinical trial for relapsed or refractory B-cell malignancies. This designation provides incentives to encourage drug development for rare diseases.

July 02, 2025 — Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company, has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its investigational drug bexobrutideg (NX-5948) to treat lymphoplasmacytic lymphoma, also known as Waldenström macroglobulinemia (WM). This designation provides significant incentives to encourage the development of treatments for rare diseases.

Bexobrutideg is an orally bioavailable, brain penetrant degrader of Bruton’s tyrosine kinase (BTK), currently being evaluated in a Phase 1a/b clinical trial for relapsed or refractory B-cell malignancies. The EMA's Orphan Drug Designation program grants orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases affecting fewer than 5 in 10,000 people in the European Union. This designation includes 10 years of market exclusivity in the EU upon approval, access to protocol assistance, eligibility for centralized marketing authorization, and significant reductions in regulatory fees [1].

"The EMA’s Orphan Drug Designation for bexobrutideg 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."

Waldenström macroglobulinemia is a rare type of blood cancer that affects B lymphocytes, a form of white blood cell. In this disease, B cells grow and survive abnormally, leading to their accumulation in the bone marrow, lymph nodes, and spleen. Early symptoms often include fatigue and weakness. It is characterized by the overproduction of IgM paraprotein—a blood protein that can cause the blood to thicken and lead to complications such as vision issues, heart problems, hemolytic anemia, and neurological symptoms.

Bexobrutideg previously received the U.S. Food and Drug Administration’s Fast Track designation in December 2024 for the treatment of WM and Fast Track designation in January 2024 for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) after at least two lines of therapy, including a BTK inhibitor and a B-cell lymphoma 2 (BCL2) inhibitor. In November 2024, the EMA granted PRIME designation to bexobrutideg for the treatment of adult patients with relapsed or refractory CLL/SLL after treatment with at least a BTK inhibitor and a BCL2 inhibitor [2].

References:
[1] https://www.globenewswire.com/news-release/2025/07/07/3110913/0/en/European-Medicines-Agency-Grants-Bexobrutideg-NX-5948-Orphan-Drug-Designation-for-the-Treatment-of-Lymphoplasmacytic-Lymphoma-also-Known-as-Waldenstr%C3%B6m-Macroglobulinemia.html
[2] https://www.stocktitan.net/news/NRIX/european-medicines-agency-grants-bexobrutideg-nx-5948-orphan-drug-2exb2r2mjwxa.html