The eRulemaking Program
10/26/2026 | Press release | Distributed by Public on 10/26/2026 06:48
Issuance of Priority Review Voucher; Rare Pediatric Disease Product; ISEMBYLD (apitegromab-mstn)
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2026-N-11848]
Issuance of Priority Review Voucher; Rare Pediatric Disease Product; ISEMBYLD (apitegromab-mstn)
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) 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, [email protected].
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.
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 ISEMBYLD (apitegromab-mstn), go to the "Drugs@FDA" website at https://www.accessdata.fda.gov/scripts/cder/daf/.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-21718 Filed 10-23-26; 8:45 am]
BILLING CODE 4164-01-P
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