Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in AL Amyloidosis
08 Feb 2024
Orphan DrugCell TherapyImmunotherapyClinical Study
European Orphan Drug Designation (“ODD”) qualifies NXC-201 for:
10 years of market exclusivity once authorized in the EU
Access to the EU centralized authorization procedure
Reduced fees for EU protocol assistance, marketing authorization applications, inspections before authorization, applications for changes to marketing authorizations made after approval, and reduced annual fees
U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023
Feb. 07, 2024 -- Immix Biopharma, Inc. (“Immix Biopharma”, “Company”, “We” or “Us”, Nasdaq:IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, today announced that the European Commission (EC) has granted orphan drug designation to NXC-201 for the treatment of AL Amyloidosis.
“European Union orphan drug designation for NXC-201 represents an important milestone in our global strategy,” said Ilya Rachman, MD PhD, Chief Executive Officer, Immix Biopharma. “We believe we are addressing an urgent need to establish a new potential one-time treatment option for relapsed/refractory AL Amyloidosis patients.” Gabriel Morris, Chief Financial Officer, Immix Biopharma, added, “We believe NXC-201’s observed favorable tolerability profile and ‘Single Day CRS’ across a robust clinical dataset to-date enables our observed complete responses (ongoing) in heavily pretreated relapsed/refractory AL Amyloidosis patients and potential expansion into other autoimmune diseases.”
Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease.
About Immix Biopharma, Inc.
Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease. Our lead cell therapy asset is next generation CAR-T NXC-201 for autoimmune disease, relapsed/refractory AL Amyloidosis, and relapsed/refractory multiple myeloma, currently being evaluated in our ongoing Phase 1b/2a NEXICART-1 (NCT04720313) clinical trial. NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T (CRS median onset day 1, median duration 1 day), enabling the potential for a faster return home for patients and supporting ongoing expansion into autoimmune indications. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma. Our second program, a tissue specific therapeutic (TSTx) asset IMX-110, is being evaluated in Phase 1b/2a clinical trials as a monotherapy and IMMINENT-01 combination clinical trial with BeiGene’s anti-PD-1 antibody tislelizumab in relapsed/refractory solid tumors. IMX-110 has been awarded Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the FDA. Learn more at www.immixbio.com.
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