CARsgen Therapeutics Receives Positive EMA Opinion on Orphan Drug Designation for Fully Human Anti-BCMA CAR-T Cells (CT053) for the Treatment of Multiple Myeloma

On April 27, 2020 CARsgen Therapeutics Co., Ltd., a clinical-stage biopharmaceutical company, reported the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on CARsgen’s application for orphan designation of its investigational CT053 CAR T-cell therapy, fully human anti-BCMA (B cell maturation antigen) autologous chimeric antigen receptor (CAR) T cells for the treatment of multiple myeloma (Press release, Carsgen Therapeutics, APR 27, 2020, View Source [SID1234556661]). CT053 was previously granted orphan drug designation by the US Food and Drug Administration in August 2019.

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"The COMP’s adoption of a positive opinion for CT053 orphan drug designation is another important recognition of CARsgen’s commitment to the development of its potential best-in-class CAR T cell therapy for the treatment of multiple myeloma," said Zonghai Li, M.D., Ph.D., Chief Executive Officer of CARsgen. "We look forward to working closely with the EMA under the recently granted PRIME eligibility and continuing the clinical development of CT053 in Europe."

The positive opinion issued by COMP will be sent to the European Commission, which is expected to grant the orphan designation within 30 days. EMA orphan drug designation helps support the development of new treatments for rare conditions, those affecting not more than five in 10,000 people in the EU, that are life-threatening or chronically debilitating. Medicines that meet the EMA’s orphan designation criteria qualify for incentives to encourage advancement of drug development.