Roche Holding AG — Evrysdi FDA Review (NDA219285, ORIG 1, 2025)

Roche Holding AG · 2025 · FDA Review (openFDA) · company profile →

Report summary

This report summarizes the FDA's clinical review of Roche Holding AG's Evrysdi (risdiplam) 5-mg film-coated tablet, a new formulation for spinal muscular atrophy (SMA). The review primarily focuses on establishing bioequivalence between this new tablet and the previously approved oral solution, rather than evaluating new efficacy or safety data. The FDA's PDUFA goal date for this application is February 11, 2025. The report recommends approval of the tablet for the treatment of SMA in both pediatric and adult patients.

Key findings — cited to the page

Application Number	219285	EVRYSDI Film-Coated Tablets · p.2
PDUFA Goal Date	February 11, 2025	EVRYSDI Film-Coated Tablets · p.2
Recommended Regulatory Action	Approve	EVRYSDI Film-Coated Tablets · p.2
Recommended Indication	Treatment of spinal muscular atrophy (SMA) in pediatric and adult patients	EVRYSDI Film-Coated Tablets · p.2
Original Evrysdi Oral Solution Approval Date	August 7, 2020	EVRYSDI powder for oral solution · United States · p.10
Evrysdi Oral Solution Indication Extension Approval Date	27 May 2023	EVRYSDI powder for oral solution · p.10
Evrysdi Oral Solution USPI Update Approval Date	September 17, 2024	EVRYSDI powder for oral solution · p.10
Evrysdi Oral Solution International Approvals	over 60 markets	EVRYSDI powder for oral solution · Global · p.10
Number of healthy volunteers in Study BP42066	124 subjects	Study BP42066 · p.12
Discontinuations due to TEAEs	6 subjects	Study BP42066 · p.14
TEAEs in Part 1 of Study BP42066	17 events	Study BP42066 · p.14
Subjects with TEAEs in Part 1 of Study BP42066	13 subjects	Study BP42066 · p.14

Source: Roche Holding AG ↗ · License: US Government public domain. Summary and findings are extracted from the published document; every figure cites its page. Informational only — not investment advice. Explore the Neurology library →