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 deflazacort (Emflaza®, PTC Therapeutics) and 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.

For questions, please contact Catherine Koola, Program Manager, at ckoola@icer-review.org.

Associated Meetings

July 25, 2019 10am-4pm ET

MIT Samberg Conference Center
Salon I/T
50 Memorial Drive
Cambridge, MA 02142

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

05/22/2019

05/22/2019

05/22/2019 – 06/18/2019

07/11/2019

07/11/2019

07/11/2019

07/25/2019

07/25/2019
Evidence Presentation

08/15/2019
Final Evidence Report and Meeting Summary

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