RARE Daily

FDA Grants Forma Rare Pediatric Disease Designation for SCD Therapy

February 20, 2020

Rare Daily Staff

The U.S. Food and Drug Administration has granted Forma Therapeutics the Rare Pediatric Disease and Fast Track designations for FT-4202 for the treatment of sickle cell disease, a progressive and life-threatening inherited disease.

Sickle cell disease (SCD) is a red blood cell disorder that causes a patient’s oxygen-carrying cells to be abnormally inflexible and sickle-shaped. This genetic defect leads to lowered oxygen-carrying capacity in the hemoglobin protein of these red blood cells. Sickle cell disease can cause inflammation of extremities, bacterial infections, stroke, anemia, and attacks of pain called vaso-occlusive crises.

FT-4202 is a selective pyruvate kinase-R activator that exhibits the potential to beneficially impact both anemia and vaso-occlusive crises for people living with SCD. It works by helping hemoglobin hold on to oxygen molecules longer and to reduce red blood cell sickling. Forma is currently enrolling patients with SCD in a phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics of FT-4202. Forma plans to initiate a registrational trial within the next year.

“These designations for Forma’s lead clinical asset underscore the FDA’s continued recognition of the needs of patients with SCD,” said Frank Lee, CEO of Forma Therapeutics. “With more than 100,000 individuals living with SCD in the U.S., and with relatively few treatment options, we believe significant unmet medical needs persist. These FDA designations give Forma the opportunity to accelerate the development of a new treatment for people impacted by SCD.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes ARU1801 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its Rare Pediatric Disease voucher to Vifor Pharma $111 million in February 2020.

Photo: Frank Lee, CEO of Forma Therapeutics


Editor’s note: This story was updated to correct the most recent sales of a rare pediatric disease voucher.

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