Miplyffa : Withdrawal of the marketing authorisation application



Orphazyme A/S withdrew its application for a marketing authorisation of Miplyffa for the treatment of Niemann-Pick disease type C (NPC) in patients aged 2 years and older in combination with miglustat (another medicine to treat NPC).

The company withdrew the application on 22 March 2022.

  • List item

    Questions and answers on the withdrawal of application for the marketing authorisation of Miplyffa (arimoclomol) (PDF/115.83 KB)

    First published: 22/04/2022

  • Key facts

    Product number
    International non-proprietary name (INN) or common name
    • arimoclomol
    Active substance
    • arimoclomol citrate
    Date of withdrawal
    Company making the application
    Orphazyme A/S
    Withdrawal type
    Initial authorisation

    All documents

    Related information on withdrawals

    A question-and-answer (Q&A) document provides a summary of the CHMP's evaluation of the medicine at the time of the withdrawal of the application, and includes a link to the company's formal withdrawal letter.

    An assessment report is published when the application is withdrawn after the first stage of the CHMP's evaluation is completed ('day 120').

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