Amondys 45 And Vyondys 53 One Step Closer To Traditional Approvals

研报英文原文证据摘录

Amondys 45 And Vyondys 53 One Step Closer To Traditional Approvals

Quick Note

June 30, 2026 Sarepta Therapeutics (SRPT)

Rating: Amondys 45 And Vyondys 53 One Step Closer To TraditionalOUTPERFORM ApprovalsPrice:

$17.02

12-Month Price Target: SRPT announced this morning that the FDA has accepted for filing the sNDAs

$35.00 to convert Amondys 45 and Vyondys 53's accelerated approvals to traditional

approvals, and the PDUFA date will be 2/28/2027. We see the FDA's decision as

an important step forward since it confirms that SRPT has submitted adequate

Analysts data, including both results from the randomized, double-blind, placebo-controlled

Yun Zhong, Ph.D. Ph3 ESSENCE confirmatory study and real-world evidence, to support a review.(917) 608-0166

yun.zhong@wedbush.com We expect the FDA to recognize Amondys 45 and Vyondys 53's favorable clinical

profiles, and we remain optimistic in both therapies receiving traditional approvals

by the PDUFA data to continue benefiting amenable DMD patients.

ESSENCE Study Did Show Efficacy. Recall ESSENCE enrolled an ITT population of

225 patients, and data from the casimersen cohort supported the FDA approval

for Amondys 45 in Feb. '21. Patients were 2:1 randomized to receive Vyondys 53

(n=58) or Amondys 45 (n=90) at 30mg/kg, or placebo (n=77), for 96 weeks. The

primary endpoint was initially 6-minute walk test (6MWT) but became change from

baseline in the 4-step ascend velocity at week 96 after a protocol modification in

Dec. '24. Top-line data from the study in Nov. '25 showed numerically differences

in both primary endpoint and secondary endpoint measures that favored Vyondys

53 and Amondys 45, although no statistical significance was reached in any of

the endpoints. However, a post hoc analysis that excluded 23 patients whose