Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in AL Amyloidosis
The orphan drug designation (ODD) granted by the European Commission to NXC-201 for the treatment of AL Amyloidosis is a significant regulatory milestone for Immix Biopharma. This status is particularly important for therapies targeting rare diseases, as it provides a range of incentives designed to support drug development. These include market exclusivity for a decade post-authorization, which can protect the company's investment in the drug by reducing competition. Additionally, reduced regulatory fees and access to the centralized authorization procedure can lower the financial barriers to market entry and expedite the approval process.
In the context of AL Amyloidosis, a rare and life-threatening condition, the ODD could facilitate Immix Biopharma's efforts to bring a new treatment option to market. The company's focus on a 'Single Day CRS' protocol and the potential for NXC-201's application in other autoimmune diseases could further amplify its market impact. The increasing prevalence of relapsed/refractory AL Amyloidosis, growing at an annual rate of 12%, underscores the urgency and potential market for such treatments.
The grant of ODD to NXC-201 in the EU could be a precursor to increased investor confidence in Immix Biopharma's stock. Investors often view regulatory milestones as indicators of a company's progress and potential for future revenue growth. The designation suggests that NXC-201 has a unique treatment proposition for a growing patient population estimated to reach nearly 30,000 in 2023. The financial implications include not only the potential revenue from the drug's sales but also the cost savings associated with reduced regulatory fees and streamlined approval processes.
Furthermore, the company's ability to potentially expand the drug's application to other autoimmune diseases could lead to a diversification of its product pipeline , reducing the risk profile of the company's portfolio. This diversification strategy could attract a broader range of investors and positively influence the company's valuation in the stock market.
The economic implications of ODD for a drug like NXC-201 extend beyond Immix Biopharma's financials. From a healthcare economics perspective, the cost-effectiveness of new treatments for rare diseases is critical. The incentives provided by the ODD, such as market exclusivity, can help offset the high costs of research and development for drugs that serve a small patient population. This is particularly relevant in the EU, where healthcare systems are often publicly funded and cost containment is a priority.
Moreover, the potential for NXC-201 to serve as a one-time treatment option could lead to long-term savings for healthcare systems by reducing the need for ongoing treatment and management of relapsed/refractory AL Amyloidosis. The societal impact of introducing effective treatments for rare diseases also includes improved patient outcomes and quality of life, which are important considerations for healthcare policy makers.
02/07/2024 - 09:37 AM
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
Los Angeles, Feb. 07, 2024 (GLOBE NEWSWIRE) -- 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 .
Forward Looking Statements
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What is the ticker symbol for Immix Biopharma, Inc.?
The ticker symbol for Immix Biopharma, Inc. is IMMX.
What is the European Orphan Drug Designation for NXC-201?
The European Orphan Drug Designation provides 10 years of market exclusivity in the EU, access to the centralized authorization procedure, and reduced fees for various regulatory processes.
What is the estimated prevalence of relapsed/refractory AL Amyloidosis in the U.S.?
The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing at 12% per year, estimated to reach 29,712 patients in 2023.
What are the benefits of orphan drug designation 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.
