On July 7, 2025 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted protein degradation medicines, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to bexobrutideg (NX-5948) for the treatment of lymphoplasmacytic lymphoma (Press release, Nurix Therapeutics, JUL 7, 2025, View Source [SID1234654261]). Bexobrutideg is an orally bioavailable, brain penetrant degrader of Bruton’s tyrosine kinase (BTK) which is being evaluated in an ongoing Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies, including lymphoplasmacytic lymphoma, also known as Waldenström macroglobulinemia (WM).
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The EMA’s Orphan Drug Designation program grants orphan status to therapies intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 5 in 10,000 people in the European Union. This designation provides several incentives to encourage the development of treatments for rare conditions, including 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.
"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.
About Bexobrutideg (NX-5948)
Bexobrutideg is an investigational, orally bioavailable, brain penetrant, small molecule degrader of BTK currently being evaluated in a Phase 1 clinical trial in patients with relapsed or refractory B cell malignancies. Nurix previously has reported encouraging safety and efficacy data in patients with WM treated in the ongoing Phase 1a/b clinical trial of bexobrutideg, demonstrating early promise of clinical benefit with potential for durable outcomes. Nurix continues to enroll patients with WM in an ongoing Phase 1b expansion cohort and anticipates sharing additional clinical data in 2025. Additional information on the ongoing clinical trial can be accessed at clinicaltrials.gov (NCT05131022). Nurix is also developing bexobrutideg for the potential treatment of inflammatory diseases.