2021-05208. Issuance of Priority Review Voucher; Rare Pediatric Disease Product

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice.

SUMMARY:

The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act), as amended by the Food and Drug Administration Safety and Innovation Act (FDASIA), authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that AMONDYS 45 (casimersen), manufactured by Sarepta Therapeutics Inc., meets the criteria for a priority review voucher.

FOR FURTHER INFORMATION CONTACT:

Althea Cuff, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-4061, Fax: [phone], email: [e-mail].

SUPPLEMENTARY INFORMATION:

FDA is announcing the issuance of a priority review voucher to the sponsor of an approved rare pediatric disease product application. Under section 529 of the FD&C Act (21 U.S.C. 360ff), which was added by FDASIA, FDA will award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined that AMONDYS 45 (casimersen) manufactured by Sarepta Therapeutics Inc., meets the criteria for a priority review voucher. AMONDYS 45 (casimersen) is indicated for the treatment of Duchenne Muscular Dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

For further information about the Rare Pediatric Disease Priority Review Voucher Program and for a link to the full text of section 529 of the FD&C Act, go to https://www.fda.gov/​ForIndustry/​DevelopingProductsforRareDiseasesConditions/​RarePediatricDiseasePriorityVoucherProgram/​default.htm. For further information about AMONDYS 45 (casimersen), go to the “Drugs@FDA” website at https://www.accessdata.fda.gov/​scripts/​cder/​daf/​.

Dated: March 9, 2021.

Lauren K. Roth,

Acting Principal Associate Commissioner for Policy.