Forma Therapeutics Holdings (FMTX) Announces FT-4202 Receives Orphan Drug Designation in Europe for Treatment of Sickle Cell Disease

November 10, 2020 7:16 AM EST
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Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced the European Commission granted Orphan Drug designation to Forma’s FT-4202 for the treatment of sickle cell disease (SCD), based on a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA). FT-4202 was previously granted Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of patients with SCD.

“I am pleased to see the regulatory community recognize the urgent need to bring therapies to people living with sickle cell disease,” said Frank Lee, president and chief executive officer of Forma. “Without effective treatment, sickle cell can affect all organs over time and lead to substantial suffering for those born with this genetic disease. We embrace the potential this designation may provide as we prepare to initiate a global registrational Phase 2/3 trial with FT-4202 in the first quarter of 2021.”

Forma is currently enrolling patients with SCD in a randomized, placebo-controlled, multi-center Phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202. For more information on eligibility and study sites for the open Phase 1 study, please visit

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