On March 31, 2022 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, reported that the European Medicines Agency (EMA) has granted obe-cel, Autolus’ leading CAR T clinical candidate, Orphan Medical Product Designation for treatment of acute lymphoblastic leukemia (ALL) patients (Press release, Autolus, MAR 31, 2022, View Source [SID1234611309]).
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"This regulatory designation is an important milestone toward addressing the urgent, unmet need in ALL patients, and follows the Orphan Drug Designation previously granted in 2019 by the U.S. Food and Drug Administration (FDA) for obe-cel," said Dr. Christian Itin, Chief Executive Officer of Autolus. "Recruitment is ongoing in the Phase 2 portion of the pivotal study of obe-cel and we look forward to announcing first Phase 2 data this year."
Orphan Medical Product Designation is granted by the EMA Committee for Orphan Medicinal Products to medicines intended for the treatment, diagnosis or prevention of a disease that is life-threatening or chronically debilitating that affects fewer than 5 in 10,000 people in the EU under the Orphan Regulation (Regulation (EC) No 141/2000). To attain Orphan Designation, it must be justified that a medicinal product could potentially be of significant benefit compared to the existing authorized medicinal products for those affected by the condition. Benefits of the designation include fee reductions for centralized activities including; applications for marketing authorization, inspections and scientific advice. A key benefit is ten years of market exclusivity in the EU following marketing approval by the EMA.