Tofersen receives positive opinion from European Medicines Agency Committee

We welcome the positive opinion on tofersen (Qalsody) by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and what this means for patient access in Europe.

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced a positive opinion for the approval of tofersen for the treatment of the SOD1 motor neuron disease (MND). The CHMP panel reviews the scientific evidence and forms an opinion on whether new drugs should be approved in Europe. The committee based their review on data from the phase 3 VALOR trial and its open label extension.

The EMA has recommended to the European Commission (EC) that tofersen is given marketing authorisation for treating people with the SOD1 type of MND in the European Union. The CHMP’s recommendation will be reviewed by the EC with a final decision expected in the second quarter of 2024. A positive EC decision would mean that tofersen is approved as the first treatment for the SOD1 type of MND across all 27 European Union member states, as well as Iceland, Liechtenstein, Norway, and Northern Ireland.

The three MND charities in the UK – My Name’5 Doddie Foundation, MND Association and MND Scotland – will continue to work together to understand this outcome and what it means for people with the SOD1 type of MND in the UK, and our next steps.

More about Tofersen and what this means for the UK

Tofersen has been developed by Biogen for use in people with the SOD1 type of MND. SOD1-MND is a rare genetic subtype of MND, accounting for 2% of total MND cases.

Everyone has the SOD1 gene and it contains the instructions for making an enzyme important for keeping the cells of our bodies healthy. In people with SOD1-MND, the DNA code for SOD1 contains a mistake, meaning that their cells can’t make this enzyme properly and instead makes a version that is damaging.

Tofersen stops the faulty SOD1 code from being used to make SOD1 protein, reducing the amount of toxic material being made.

In April 2023, tofersen was approved in the USA for treating people with the SOD1 type of MND. This news from the EMA means it may be made available to people in Europe (depending on final authorisation by the EC).

At the moment, tofersen is not approved for use in the UK. Biogen, the company that has created it, has provided the following update:

Following Brexit, marketing authorisations for medicines in Great Britain (England, Scotland and Wales) are determined by the Medicines and Healthcare products Regulatory Agency (MHRA). We are in dialogue with the MHRA, National Institute for Health and Care Excellence (NICE) and the Scottish Medicines Consortium (SMC) on the most appropriate access route for tofersen in the UK.