RARE Daily

FDA Grants Seelos Therapeutics Rare Pediatric Disease Designation for Sanfilippo Syndrome Therapy

May 15, 2020

The U.S. Food and Drug Administration granted Seelos Therapeutics Rare Pediatric Disease designation for SLS-005 in Sanfilippo syndrome, a rare, lysosomal storage disorder.

Sanfilippo syndrome, also known as MPS III, is a genetic disease that causes a deficiency of an enzyme need to break down metabolic waste. In the absence of adequate levels of the enzyme, waste materials accumulate in the cells of the body and cause progressive damage. It can cause delayed development, behavioral problems, sleep difficulties, joint problems, and other issues.

SLS-005 is a low molecular weight disaccharide that protects against pathological processes in cells. It has been shown to penetrate muscle and cross the blood brain barrier. Seelos was recently granted Orphan Disease designation from the FDA for SLS-005 in Sanfilippo syndrome.

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 SLS-005 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

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.

Editor’s note: This story was updated to correct the most recent sales of a Rare Pediatric Disease voucher.

Author: Rare Daily Staff

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