2020-20320. Issuance of Priority Review Voucher; Rare Pediatric Disease Product

AGENCY:

Food and Drug Administration, Health and Human Services (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 TRIKAFTA (elexacaftor/tezacaftor/ivacaftor), manufactured by Vertex Pharmaceutical, 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 TRIKAFTA (elexacaftor/tezacaftor/ivacaftor), manufactured by Vertex Pharmaceutical, Inc., meets the criteria for a priority review voucher.

TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) is indicated for the treatment of patients with cystic fibrosis aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

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 TRIKAFTA (elexacaftor/tezacaftor/ivacaftor), go to the “Drugs@FDA” website at https://www.accessdata.fda.gov/​scripts/​cder/​daf/​.

Dated: September 9, 2020.

Lowell J. Schiller,

Principal Associate Commissioner for Policy.