Duchenne Muscular Dystrophy

New England CEPAC

ICER will assess the comparative clinical effectiveness and value of treatments for Duchenne muscular dystrophy. This evidence review will examine two exon-skipping therapies: eteplirsen (Exondys 51®, Sarepta) and golodirsen (Sarepta). Eteplirsen was approved by the FDA in September 2016 for patients with a confirmed mutation in the dystrophin gene that is amenable to exon 51 skipping. Sarepta has announced its intention to file for accelerated approval of golodirsen by the end of 2018. This list is tentative and subject to change.

Associated Meetings

July 25, 2019 10am-4pm ET

Boston, MA

The New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.


Key Dates

Associated Materials

12/13/2018 – 01/08/2019
Open Input Period

01/11/2019

01/11/2019 – 02/01/2019

01/11/2019

02/11/2019

03/19/2019

04/09/2019
Model Analysis Plan

05/22/2019
Draft Evidence Report

05/22/2019
Draft Evidence Report

05/22/2019 – 06/18/2019
Public Comments

07/11/2019
Evidence Report

07/11/2019
Revised Voting Questions

07/11/2019
Response to Comments

07/25/2019
Meeting Agenda

07/25/2019
Evidence Presentation

08/15/2019
Final Evidence Report and Meeting Summary

08/15/2019
Report-at-a-Glance