On April 29, 2024 Immix Biopharma, Inc. ("Immix Biopharma", "Company", "We" or "Us", Nasdaq:IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, reported that the European Commission (EC) has granted orphan drug designation to NXC-201 for the treatment of multiple myeloma (Press release, Immix Biopharma, APR 29, 2024, View Source [SID1234642427]).
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"Frail patients, heavily represented in our NEXICART-1 clinical trial, remain an area of unmet medical need and are a significant portion of the relapsed/refractory multiple myeloma population," said Ilya Rachman, MD PhD, Chief Executive Officer, Immix Biopharma. "We believe EU orphan drug designation for NXC-201 affirms the potential clinical impact of NXC-201 in this sizable population."
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 could enable an attractive option for frail relapsed/refractory multiple myeloma patients in addition to our lead indication, relapsed/refractory AL Amyloidosis, and our active NXC-201 expansion into other autoimmune diseases."
According to Davis et al., 2023 Transplantation and Cellular Therapy, commercial CAR-Ts produced 6.9 months median progression free survival in frail relapsed/refractory multiple myeloma patients in a real-world setting. 61% of patients in the dataset were considered frail.
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.