Paul-Ehrlich-Institut

Go to:

Information on the Use of Cookies

In order to operate and optimise our website, we would like to collect and analyse statistical information completely anonymously. Will you accept the temporary use of statistics cookies?

You can revoke your consent at any time in our privacy policy.

OK

Compassionate Use Programme for Onasemnogene abeparvovec-xioi for the Treatment of Spinal Muscular Atrophy Accepted

The Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, agreed on February 3, 2020 that Onasemnogene abeparvovec-xioi (in the USA: ZOLGENSMA) will be made available by the pharmaceutical company AveXis as part of a compassionate use programme. The gene therapy product has already received a marketing authorisation in the USA and is currently undergoing the centralised marketing authorisation procedure in Europe. Onasemnogene abeparvovec-xioi is designed for the treatment of certain forms of spinal muscular atrophy in children aged up to two years. One in 10,000 children in Germany is affected by this serious disorder of the spinal cord, which leads to muscle atrophy and in severe cases, if left untreated, can lead to death.

DNA Helix (Source: Qimono/Pixabay.com)

The medicinal preparation Onasemnogene abeparvovec-xioi from the manufacturer AveXis Europe Ltd has been authorised in the United States since May 2019. It is applied as a one-off intravenous infusion. The application for a marketing authorisation in the EU by means of the centralised procedure is currently in the evaluation phase at the European Medicines Agency (EMA). In this authorisation procedure, the Paul-Ehrlich-Institut has assumed the co-rapporteurship at the Committee for Advanced Therapies (CAT) at the EMA.

On 24 January 2020, the pharmaceutical company AveXis requested a compassionate use programme for Onasemnogene abeparvovec-xioi at the Paul-Ehrlich-Institut. Currently, a treatment satisfactory for all cases of spinal muscular atrophy in severely or life-threateningly ill children of up to two years with an already authorised medicinal product is not available in Germany. The Paul-Ehrlich-Institut assessed the manufacturer’s request and established that the compassionate use programme can start in early February 2020 from the regulatory point of view. There is no reason for rejecting the compassionate use programme or, pursuant to Section 4b (5) AMHV (Arzneimittel-Härtefall-Verordnung, German Compassionate Use Regulation), to deny the release approval of the genetically modified organism Onasemnogene abeparvovec-xioi to the company.

The compassionate use programme for Germany will end as soon as Onasemnogene abeparvovec-xioi obtains authorisation in Europe. The treating physician in certified neuropaediatric centers can register patients for participation in the compassionate use programme of the pharmaceutical company AveXis.

Background – Compassionate Use Programme

A compassionate use programme allows a new medicinal product, which has not yet been granted a marketing authorisation, to be made available to a group of patients under particular circumstances.

Compassionate use programmes can be applied in the event of serious or life-threatening diseases for which there is no other satisfactory treatment. Pursuant to Section 2 AMHV, prerequisites for a compassionate use programme include:

  • The patient suffers from a life-threatening disease or a disease that will lead to severe disability.
  • The patient cannot be treated satisfactorily with a medicinal product authorised in Germany and his or her participation in an on-going clinical trial in Germany is not possible.
  • An authorised clinical trial is currently conducted in Germany with the intended indication, or an application for a marketing authorisation with the intended indication has been submitted in Germany or another member state of the EU/EEA or at the EMA.

The compassionate use programme must be requested at the competent senior federal authority pursuant to Section 3 AMHV before it can be carried out. The implementation of the compassionate use programme can only be started once the competent senior federal authority, in this case the Paul-Ehrlich-Institut, has confirmed the request and, at the same time, has not rejected it (Section 4 AMHV).

Updated: 03.02.2020