FDA Grants Rare Pediatric Disease Status to Cartesian Therapeutics' Descartes-08 for Juvenile Dermatomyositis

Cartesian Therapeutics, Inc., a biotechnology firm specializing in mRNA cell therapy for autoimmune diseases, has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to its leading mRNA cell therapy candidate, Descartes-08, for the treatment of juvenile dermatomyositis (JDM). Descartes-08 is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product that targets B-cell maturation antigen (BCMA). Unlike traditional therapies, Descartes-08 does not require preconditioning chemotherapy and avoids the use of integrating vectors.

Dr. Carsten Brunn, President and CEO of Cartesian, expressed satisfaction with the FDA's decision, emphasizing the potential of Descartes-08 as a significant therapeutic option for juvenile patients with this rare condition. He reiterated the company's commitment to leveraging their novel mRNA platform to extend the benefits of cell therapy to individuals suffering from autoimmune diseases. Cartesian plans to file an Investigational New Drug (IND) application for a Phase 2 pediatric basket study by the end of the year, focusing on neurology and rheumatology autoimmune conditions, including JDM.

The Rare Pediatric Disease Designation is designed by the FDA to address serious and life-threatening diseases that primarily affect children under 18 and impact fewer than 200,000 people in the United States. This designation includes a voucher program, where, if Descartes-08 is approved for marketing, Cartesian might qualify for a priority review voucher. This voucher can be redeemed to expedite the review process of another marketing application for a different product.

Juvenile Dermatomyositis (JDM) is a rare autoimmune disorder in children, characterized by a distinctive skin rash and muscle inflammation that can affect multiple organ systems, including the joints, heart, lungs, kidneys, eyes, and gastrointestinal tract. Symptoms of JDM vary in severity, ranging from mild to life-threatening and include fatigue, joint pain, muscle weakness, and fever. Approximately 4,000 individuals in the United States are impacted by this condition.

Cartesian Therapeutics is advancing the field of mRNA cell therapies for autoimmune diseases. Its flagship product, Descartes-08, is currently under Phase 2b clinical development for generalized myasthenia gravis and Phase 2 development for systemic lupus erythematosus. The company also plans a Phase 2 basket trial to explore additional autoimmune indications. Another product in Cartesian’s pipeline, Descartes-15, is a next-generation, autologous anti-BCMA mRNA CAR-T therapy.

Cartesian’s innovative approach aims to transform the treatment landscape for autoimmune diseases by pioneering new therapies that leverage mRNA technology. The positive steps taken by the FDA reflect the promise of Descartes-08 and underscore the potential of mRNA cell therapies to address unmet medical needs in pediatric and adult populations alike.