Immix Biopharma, Inc. (Nasdaq: IMMX) has announced that the European Commission (EC) has granted Orphan Drug Designation (ODD) to NXC-201 for the treatment of multiple myeloma. This designation provides significant regulatory and commercial incentives for Immix Biopharma as it continues to develop this novel cell therapy.
Benefits of Orphan Drug Designation
The European Orphan Drug Designation qualifies NXC-201 for several key benefits, including:
- Market Exclusivity: 10 years of market exclusivity in the EU once the therapy is authorized.
- Centralized Authorization: Access to the EU centralized authorization procedure, streamlining the approval process.
- Reduced Fees: Reduced fees for EU protocol assistance, marketing authorization applications, inspections before authorization, and post-approval changes, as well as reduced annual fees.
Clinical Significance
According to Ilya Rachman, MD PhD, Chief Executive Officer of Immix Biopharma, frail patients, who are heavily represented in the NEXICART-1 clinical trial, represent an area of unmet medical need within the relapsed/refractory multiple myeloma population. He stated that the EU orphan drug designation for NXC-201 affirms the potential clinical impact of NXC-201 in this sizable population.
Gabriel Morris, Chief Financial Officer of Immix Biopharma, added that 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 the lead indication, relapsed/refractory AL Amyloidosis, and the active NXC-201 expansion into other autoimmune diseases.
NXC-201: A Novel CAR-T Therapy
NXC-201 is a next-generation CAR-T therapy currently being evaluated in the Phase 1b/2 NEXICART-1 (NCT04720313) clinical trial, which was initiated in February 2021. Immix Biopharma believes that NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T, with a median onset of CRS on day 1 and a median duration of 1 day. This rapid resolution of CRS could enable a faster return home for patients and support ongoing expansion into autoimmune indications.
Multiple Myeloma and Unmet Needs
Multiple myeloma is a cancer that forms in plasma cells, a type of white blood cell. The disease is characterized by the accumulation of abnormal plasma cells in the bone marrow, which can lead to various complications, including bone damage, anemia, kidney problems, and infections. Relapsed/refractory multiple myeloma refers to cases where the disease returns after treatment or does not respond to initial therapy.
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.
The orphan drug designation in the European Union is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify, a therapy must be intended for the treatment, prevention, or diagnosis of a life-threatening or chronically debilitating disease that affects no 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.
