The FDA has extended its review period for Nippon Shinyaku's deramiocel by 3 months, moving the PDUFA date to 22 November. The extension follows the FDA's acceptance of Phase 3 data additionally submitted by Capricor Therapeutics as a major amendment to the application, potentially affecting the launch timing and outlook for first-year sales.
Nippon Shinyaku announced that the FDA's target action date for its review of deramiocel, the PDUFA date, had been extended by 3 months from 22 August to 22 November. The extension follows the FDA's acceptance of additional Phase 3 clinical data and analysis results submitted by Capricor Therapeutics as a major amendment to the application.
Twenty-four-month open-label extension data from the HOPE-3 trial, along with additional robustness analyses, were submitted to the FDA in support of a request to refine deramiocel's indication. HOPE-3 has upper-limb function as its primary endpoint, and its data are currently regarded as among the leading clinical datasets for evaluating upper-limb function in DMD.
Nippon Shinyaku's earnings forecast for the fiscal year ending March 2027 includes ¥11.4 billion in U.S. sales of deramiocel. The review extension may affect the launch timing and the timing of first-year revenue recognition. The company said it has not decided to revise its full-year forecast at this stage.
In the U.S. market, Capricor Therapeutics' share price surged following the announcement of the extension. The FDA's acceptance of the additional data as a major amendment is also being viewed as a positive development toward refining the indication. The focus will now be on the outcome of the review of the additional data and the publication of Nippon Shinyaku's earnings outlook.