Seelos Receives Rare Pediatric Disease Designation for Trehalose in Sanfilippo Syndrome

Seelos Therapeutics has been granted Rare Pediatric Disease Designation (RPDD) for SLS-005 in Sanfilippo syndrome from the U.S. Food and Drug Administration (FDA).

RPDD is an incentive program created under the Federal Food, Drug, and Cosmetic Act (FD&C Act) created to encourage the development of new therapies for the prevention and treatment of certain rare pediatric diseases.

Seelos was also recently granted Orphan Disease Designation from the FDA for SLS-005 in Sanfilippo syndrome.

Under section 529 of the FD&C Act, the FDA will award priority review vouchers (PRVs) to sponsors of certain rare pediatric disease product applications that meet the criteria specified in that section.

The PRV entitles the holder to utilize the voucher alone or in conjunction with addition incentive programs to obtain priority review for a subsequent human drug application. The subsequent application would not have to meet the requirements for a priority review, the voucher may be utilized by the entity who is granted the voucher and, is also transferrable and may be sold to another entity. Transfer of the voucher requires FDA notification within 30 days of the transfer of ownership.