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, ...
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,
( printed page 67322)
Drug, and Cosmetic Act (FD&C Act) 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 ISEMBYLD (apitegromab-mstn), approved September 11, 2026, manufactured by Scholar Rock, Inc., meets the criteria for a priority review voucher.
FOR FURTHER INFORMATION CONTACT:
Quyen Tran, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-2771,
Quyen.Tran1@fda.hhs.gov.
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), FDA will award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined ISEMBYLD (apitegromab-mstn), manufactured by Scholar Rock, Inc., meets the criteria for a priority review voucher. ISEMBYLD (apitegromab-mstn) injection is indicated for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.
21 U.S.C. 360ff
www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/RarePediatricDiseasePriorityVoucherProgram/default.htm
www.accessdata.fda.gov/scripts/cder/daf/