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Vosoritide official document review · version 1

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Inside the official reviews

Reviewed 2026-09-16 · Preserved version 1

What did accelerated approval establish?

FDA’s 2021 review supported increased linear growth, based on annualized growth velocity, and required confirmation of benefit on final adult height. The original age threshold in that review is historical; see the separately dated U.S. label account for the labeling covered by this site. FDA PDF pp. 2, 24 ↗

More findings, study links and document coverage

What did the trial leave unanswered?

Study 111-301 showed a 1.57 cm/year adjusted growth-velocity difference at one year (95% CI 1.22–1.93). FDA did not treat that result as proof of improved final adult height or all achondroplasia complications. Longer follow-up and external-control comparisons had different evidentiary limits from the randomized trial. FDA PDF pp. 25, 26, 69, 71 ↗

How should follow-up studies be counted?

Study 111-302 extended 111-301 with open-label treatment. FDA’s historical review used it as supportive follow-up, not a second independent placebo-controlled trial. Four participants from the separate 111-205 study had reached near-final height in the original submission; that small observation did not settle the final-height question. FDA PDF pp. 55, 56, 72, 73 ↗

Read the separate U.S. label account → Label version and indication scope are preserved there; these agency reviews describe their own dated evidence.

Matched study identities

Protocol identifiers were matched to ClinicalTrials.gov records. This does not count extensions as new independent trials.

Document coverage

Selected sections read in 1 of 22 inventoried documents; 21 not yet reviewed. Reading selected sections does not establish complete appraisal of a document.

English PDF entries containing Voxzogo in the title in the retained September 15 EMA document-feed snapshot, plus selected FDA approval-package documents. Includes candidate associations, not a complete FDA inventory, national authorizations or worldwide coverage. Live EMA page requests returned HTTP 429 on September 16; fresh page reconciliation is pending. EMA inventory source ↗

Inspect documents, application identities and source pins

Page references use one-based PDF file pages, which can differ from printed page numbers. Candidate feed associations are not verified application identities.

Remaining work

  • Review the remaining EMA assessment, product-information, risk-management and change documents; reconcile historical applications separately.
  • Reconcile later FDA supplements and current postmarketing-study status. Earlier review conclusions are dated evidence, not a current approval-status substitute.
  • Complete full-text and correction appraisal of the related literature. Registry identity matching does not establish that all posted results or historical registry versions have been reviewed.

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Public beta · Selective source coverageNot medical advice · No dosing or purchasing guidance