FDA Grants RMAT Designation for Cartesian’s Cell Therapy for Myasthenia Gravis

Rare Daily Staff

The U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy designation for Cartesian Therapeutics’ Descartes-08 cell therapy for the treatment of myasthenia gravis, a rare clinically heterogeneous autoimmune disorder of the neuromuscular junction that causes disabling muscle weakness and fatigue.

Descartes-08, Cartesian’s lead product candidate, is an autologous mRNA CAR-T directed against the B cell maturation antigen (BCMA) initially being developed for the treatment of myasthenia gravis (MG).

“Receipt of RMAT designation underscores our belief that Descartes-08, our potential first-in-class mRNA CAR-T cell therapy, could serve as a meaningful addition to the MG treatment landscape,” said Carsten Brunn, president and CEO of Cartesian. “We look forward to working closely with the FDA to efficiently advance the development of Descartes-08 for this underserved population.”

In January 2024, Cartesian announced positive twelve-month follow-up data from its phase 2a study of Descartes-08 in patients with generalized MG. In this study, Descartes-08 was administered in an outpatient setting without integrating vectors or preconditioning chemotherapy, and durable depletion of autoantibodies and clinically meaningful improvements in MG severity scores during the one-year follow-up period were observed. Descartes-08 was observed to be well-tolerated, with no dose-limiting toxicities, cytokine release syndrome, or neurotoxicity.

The Company remains on track to report topline data from its phase 2b randomized, double-blind, placebo-controlled trial of Descartes-08 in patients with MG in mid-2024.

Established under the 21st Century Cures Act, a product candidate is eligible for RMAT designation if it is a regenerative medicine therapy intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Receiving RMAT designation offers sponsor companies the benefits of the fast track and breakthrough therapy designation programs, allowing for early, close, and frequent interactions with the FDA with the goal of expediting drug development.

Photo: Carsten Brunn, president and CEO of Cartesian