Amyloidosis

Midwest CEPAC

ICER will review new therapies for the treatment of hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage that is currently treated through supportive measures alone.

The review is expected to include inotersen (Akcea Therapeutics ), an antisense oligonucleotide that interferes with transthyretin production in the liver, and patisiran (OnpattroTM,Alnylam Pharmaceuticals), which inhibits transthyretin through an RNA interference pathway. Approvals are expected in October and August of 2018, respectively.

Associated Meetings

September 13, 2018 9am- 5pm CT

Chicago Marriott at Medical District/UIC

625 S Ashland Ave

Chicago, IL 60607

The Midwest CEPAC will deliberate and vote on ICER's report on treatments for hereditary transthyretin-related (hATTR) amyloidosis, a rare genetic condition characterized by nerve, heart, and eye damage that is currently treated through supportive measures alone. The Council will also review a report on therapies for prostate cancer during this meeting.


Key Dates

Associated Materials

02/16/2018 – 03/07/2018
Open Input Period

03/12/2018

Open to public comment until April 2, 2018. Click here for more information on submitting a public comment, please visit our stakeholder engagement page.


03/12/2018

03/12/2018 – 04/02/2018

04/09/2018

04/25/2018

07/20/2018

Open to public comment until August 17, 2018 at 5pm ET. Click here for more information.


07/20/2018

07/20/2018 – 08/17/2018

08/29/2018

08/29/2018

08/29/2018

09/13/2018

09/13/2018
Evidence Presentation

10/04/2018
Final Evidence Report and Meeting Summary

10/04/2018
Report-at-a-Glance