Modus Therapeutics AB, a company developing innovative treatments in high unmet medical need disease areas with a focus on sickle cell disease (SCD), has announced that sevuparin for the treatment of SCD was granted rare pediatric disease designation by the US Food & Drug Administration (FDA).
Sickle cell disease, an inherited blood disorder characterized by severe pain leading to organ damage, affects between 90,000-100,000 individuals in the U.S. Progressive organ damage limits the life expectancy of patients with SCD and lifetime medical care costs can exceed $1M per patient with an estimated $1B spent annually in the U.S.
Sevuparin is an innovative, proprietary polysaccharide drug with anti-adhesive, anti-aggregate and anti-inflammatory effects due to its multimodal mechanism of action. Sevuparin has the potential to restore blood flow and prevent further microvascular obstructions in children and adults with SCD. Children with SCD suffer from some of the most serious and life-threatening consequences of the disease including stroke, splenic sequestration and aplastic anemia. Mortality is a significant concern in children globally, with 92% of newborns dying in the first few years of life due to lack of early diagnosis.
Ellen K Donnelly, PhD, CEO of Modus Therapeutics, said: “We are very pleased with the FDA’s decision to grant a rare pediatric disease designation to sevuparin for SCD. The potential for Modus Therapeutics to be granted a Rare Disease Priority Review Voucher upon marketing approval of sevuparin could be an additional value driver for our shareholders.”
Created in 2014 by Congress, the Rare Pediatric Disease Priority Review Voucher Program provides incentive for the development of treatments for rare pediatric diseases. A drug qualifies for this category if the entire prevalence of the disease in the US is below 200,000 and the serious or life-threatening manifestations of the disease primarily affect individuals from birth to age 18.
If a drug candidate with a rare pediatric disease designation receives marketing approval in the US, the FDA may issue a Priority Review Voucher to the sponsor company. This voucher can be redeemed to provide Priority Review of a subsequent marketing application for a different product from Modus Therapeutics or another company.