FDA Grants Orphan Drug Designation to FT-4202 for Sickle Cell Treatment

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Forma TherapeuticsFT-4202 for the treatment of sickle cell disease (SCD)

This designation is given to investigational therapies intended to treat rare conditions (those that affect fewer than 200,000 people in the U.S.). It provides companies with certain financial incentives for treatment development, such as tax credits related to clinical testing, special fee exemptions, as well as marketing exclusivity in the U.S. market for seven years.

It follows the recent fast track and rare pediatric disease designations given to FT-4202 by the FDA.

“Despite newly approved therapies for SCD, a significant unmet need remains for an innovative, truly disease-modifying treatment,” Frank Lee, Forma’s CEO, said in a press release.

“Receiving Orphan Drug designation for this investigational agent, along with the formerly granted FDA Fast Track designation and Rare Pediatric Disease designation, validates the work we are doing to address the unique struggles of patients living with the rare genetic anomaly that leads to SCD,” Lee said.

FT-4202 is a small molecule intended to be a disease-modifying therapy for people with SCD. Taken orally, it works by activating a protein called pyruvate kinase-R (PKR). This protein is important for reducing the levels of 2,3-diphosphoglycerate (2,3-DPG), a byproduct of cellular metabolism that decreases the ability of hemoglobin to bind to oxygen.

By activating PKR, FT-4202 is expected to reduce levels of 2,3-DPG. That is expected to help hemoglobin hang on to oxygen more effectively and reduce the “sickling” of red blood cells that characterizes SCD.

FT-4202 also may increase levels of adenosine triphosphate (ATP) — the cellular “energy currency” — in red blood cells, improving their survival. Through these mechanisms, it is believed that FT-4202 may reduce anemia and the frequency of vaso-occlusive crises in people with SCD.

FT-4202 is being investigated in a Phase 1 clinical trial (NCT03815695), sponsored by Forma. This study is intended to assess the safety and tolerability of the medication, as well as its pharmacological profile. The trial is evaluating FT-4202 first in healthy volunteers, then in people with SCD.

Expected to conclude in September, the study is still recruiting participants at multiple locations in the U.S. Additional information is available here.

Data from the first group of healthy volunteers (which included 32 participants who received a single dose of FT-4202 or a placebo, as well as 48 participants who received multiple doses of the therapy or a placebo for two weeks), suggested that FT-4202 has a favorable safety and tolerability profile, with most reported adverse side effects being mild in severity.