Rare Daily Staff

The U.S. Food and Drug Administration granted Modus Therapeutics’ sevuparin Rare Pediatric Disease designation for the treatment of sickle cell disease, a rare genetic disease that impairs the ability of red blood cells to transport oxygen.

The Rare Pediatric Disease Priority Review Voucher Program provides incentive for the development of treatments for rare pediatric diseases. If a drug candidate with a rare pediatric disease designation receives marketing approval in the United State, the FDA may issue a Priority Review Voucher to the sponsor company. This is potentially lucrative because the voucher is transferable. In December, Ultragenyx sold a Rare Pediatric Disease Priority Review Voucher for $130 million.

Sickle cell disease causes progressive organ damage limits the life expectancy of people with the condition. Damage to organs, such as the spleen, impairs immunity and causes high risk of infections such as pneumonia. Large vessels in the brain can be affected by the rigid red blood cells and anemia, leading to overt strokes in 10-15 percent of children and silent strokes in many more. Chronic anemia also affects the heart and lungs.

Sevuparin is a proprietary polysaccharide drug with anti-adhesive, anti-aggregate, and anti-inflammatory effects due to its multimodal mechanism of action. Modus said that sevuparin has the potential to restore blood flow and prevent further microvascular obstructions in children and adults with sickle cell disease.

The Rare Pediatric Disease Priority Review Voucher Program provides incentive for the development of treatments for rare pediatric diseases. If a drug candidate with a rare pediatric disease designation receives marketing approval in the United State, the FDA may issue a Priority Review Voucher to the sponsor company. This is potentially lucrative because the voucher is transferable. In December, Ultragenyx sold a Rare Pediatric Disease Priority Review Voucher for $130 million.

“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,” Ellen Donnelly, CEO of Modus Therapeutics.

April 19, 2018
Photo: Ellen Donnelly, CEO of Modus Therapeutics

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