Decision

Early access to medicines scheme (EAMS) scientific opinion: patisiran-LNP to treat adults with hereditary transthyretin-mediated amyloidosis

Positive scientific opinion - Alnylam UK Limited for patisiran-LNP to treat adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).

From:
Medicines and Healthcare products Regulatory Agency
Published
3 August 2018
Last updated
7 August 2018 — See all updates

This publication was withdrawn on

This positive scientific opinion has been withdrawn because it has been granted an EU licence.

Documents

Patisiran-LNP- Public assessment report

PDF, 670 KB, 4 pages

Treatment protocol for patients

PDF, 195 KB, 8 pages

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Treatment protocol for healthcare professionals

PDF, 300 KB, 17 pages

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Treatment protocol on the pharmacovigilance system

PDF, 180 KB, 4 pages

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Patisiran-LNP - background information for medical directors

PDF, 60.9 KB, 2 pages

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Details

This positive scientific opinion was issued to Alnylam UK Limited for patisiran-LNP to treat adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).

The scientific opinion includes:

  • a public assessment report
  • a treatment protocol:
    • for healthcare professionals
    • for patients
    • on the pharmacovigilance system
    • EAMs scientific opinion – background information for Medical Directors

Contact

Information and details regarding patient access

For new patients wishing to access EAMS medicines in England, trusts must register patients on the NHS England web based registration system. Queries to NHS England regarding the scheme can be submitted to england.eams@nhs.net.

For information about access in Northern Ireland, contact Mark Timoney, Chief Pharmaceutical Officer at Mark.Timoney@dhsspsni.gsi.gov.uk and Joe Magee, Secondary Care Directorate at Joe.Magee@dhsspsni.gov.uk.

For information about access in Scotland contact the Scottish Government Directorate General Health & Social Care at John.Hannah@gov.scot and Rosemarie.Parr@gov.scot.

For information about access in Wales, contact the Welsh Government Department of Health and Social Services by emailing Andrew Evans, Interim Chief Pharmaceutical Officer at Andrew.Evans@wales.gsi.gov.uk and Karen Eveleigh, Head of Pharmacy and Prescribing policy at Karen.Eveleigh@wales.gsi.gov.uk.

Published 3 August 2018
Last updated 7 August 2018 + show all updates

  1. Uploaded new contact information and details regarding patient access

  2. First published.