Generic Name:
Project Status:
Therapeutic Area:
hereditary transthyretin amyloidosis
Akcea Therapeutics Inc.
Call for patient/clinician input open:
Brand Name:
Project Line:
Reimbursement Review
Project Number:
Call for patient/clinician input closed:
NOC Status at Filing:
Post NOC
Manufacturer Requested Reimbursement Criteria1:
For the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
Submission Type:
Fee Schedule:
Schedule A
hereditary transthyretin amyloidosis
Date NOC Issued:
Recommendation Type:
Reimburse with clinical criteria and/or conditions
Final Recommendation:
  1. The requested reimbursement criteria are provided by the applicant and do not necessarily reflect the views of CADTH. Reimbursement criteria from CADTH will be documented in the final recommendation, if applicable.

Key Milestones2

Call for patient input posted January 24, 2019
Patient group input closed March 15, 2019

- Patient input submission received from Hereditary Amyloidosis Canada

Patient input summary sent for review to patient input groups March 21, 2019
Patient group comments on input summary closed March 28, 2019

- No patient input summary feedback received

Submission received February 22, 2019
Submission accepted March 08, 2019
Review initiated March 11, 2019

- Selected for participation in CADTH/INESSS Clinical Engagement Pilot

Draft CADTH review report(s) sent to sponsor June 04, 2019
Comments from sponsor on draft CADTH review report(s) received June 13, 2019
Redaction requests from sponsor on draft CADTH review report(s) received June 20, 2019
CADTH review team's comments on draft CADTH review report(s) sent to sponsor July 05, 2019
Canadian Drug Expert Committee (CDEC) meeting July 17, 2019
CDEC recommendation & redacted CADTH review report(s) sent to sponsor and drug plans July 31, 2019
Embargo period ended and validation of redacted CADTH review report(s) received September 13, 2019

- Request for extension to embargo period received from the manufacturer

- Embargo extension request granted

- Reconsideration requested

Sponsor's request for reconsideration placed on CDEC agenda November 20, 2019
CDEC recommendation sent to sponsor and drug plans November 27, 2019
Embargo period ended December 11, 2019
CDEC Final Recommendation issued to sponsor and drug plans December 18, 2019
CDEC Final Recommendation posted December 20, 2019
Final CADTH review report(s) posted January 10, 2020