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FDA approves AMX0035 for the treatment of adults with MND

07 October 2022

The U.S. Food and Drug Administration (FDA) has approved AMX0035 for the treatment of adults with motor neuron disease (MND)

What is AMX0035 and how does it help people with MND?

AMX0035 (also known as RELYVRIO) is a combination of two drugs that act together to reduce the death of nerve cells by targeting two cellular processes that are impaired in MND and are thought to contribute to progression of disease. It's the first MND treatment to show a significant slowing in both disease progression and functional decline, as well as extended survival, in a randomized clinical trial. People who took the new treatment lived a median of 6.5 months longer than those taking a dummy drug.

The treatment was originally considered for FDA approval in March 2022 and received a negative vote. However, the company behind the drug, Amylyx, resubmitted new data from studies looking at the drug’s long-term effects which led to the positive FDA vote just six months later.

Today’s FDA approval of [AMX0035] is an exciting milestone for the [MND] community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx.

We want to give a heartfelt thank you to the broader [MND] community, including healthcare professionals and those living with [MND], for their guidance, support of our clinical programs, and for sharing their experiences with us. Their stories inspired us and helped our team to better understand the [MND] clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning and there is much more to be done.

This approval of a new MND treatment, only the third in the history of the disease, provides renewed hope that more treatment options are on the horizon. Whilst this approval is a very welcome step forward, AMX0035 is not a cure for MND. Continued research is therefore still urgently needed. My Name’5 Doddie Foundation is committed to funding research that will lead to the development of new and effective treatments, working towards our vision of a world free from MND.

What have the clinical trials of AMX0035 shown?

A multicentre trial of AMX0035 called the CENTAUR trial was carried out in the United States. It enrolled 137 people with MND whose symptoms had started within the previous 18 months. People already taking riluzole or edaravone were eligible to join the trial, and could continue taking these medicines throughout the trial. 89 people received AMX0035 and 48 people received a dummy drug.

The trial showed that people treated with AMX0035 for 24 weeks had a slower rate of decline compared to people treated with the dummy drug. In other words, treatment with AMX0035 slowed disease progression.

At the end of the trial, participants were invited to enroll into an “open-label extension” (OLE) that aimed to assess the long-term safety and effectiveness of AMX0035 in MND. 90 people who had enrolled in the CENTAUR trial continued into the OLE and received AMX0035 treatment; 34 of whom had been originally randomized to the dummy drug and 56 who had received AMX0035.

The results of the OLE showed that over this longer time period (up to 35 months), the people that had originally received AMX0035 in the CENTAUR study lived a median of 6.5 months longer than those who had originally received the dummy drug. Further analyses also showed that earlier administration of AMX0035 reduced the risk of hospitalisation and prolonged survival without the need for tracheostomy or permanent assisted ventilation. This encouraging data supports the potential added benefits of AMX0035 on reducing health burden in MND.

Taken together, these results demonstrate that treatment with AMX0035 significantly slows both disease progression and functional decline, and extends survival.

What does this mean for people with MND in the UK?

Although AMX0035 is now approved by the FDA for the treatment of people in the United States, it is not currently approved by the UK’s regulatory agency (the MHRA) and is therefore not yet available for the treatment of people with MND in the UK. Amylyx has submitted an application to the European Medicines Agency (EMA) which is under review. However, it is currently unclear whether this will lead to UK authorisation and when; the UK is not regulated by the EMA in the same way since leaving the EU. We will continue to share updates when new information becomes available.

In the meantime, the only way the drug can currently be accessed in the UK is through a clinical trial which is currently recruiting in Plymouth and Sheffield. Additional sites are expected to open in the UK soon. For more information and to keep updated on new sites as they open, please head to the AMX0035 page on the MND Association’s website.

Further information:

You can find out more on Amylyx’s website or by reading the published clinical trial data:

  • N Engl J Med 2020; 383:919-930. DOI: 10.1056/NEJMoa1916945.
  • Muscle Nerve 2021;63(1):31-39. DOI: 10.1002/mus.27091.
  • J Neurol Neurosurg Psychiatry 2022;93(8):871-875. DOI: 10.1136/jnnp-2022-329024.

If you have further questions, you can get in touch with our team at research@myname5doddie.co.uk

FDA approves AMX0035 for the treatment of adults with MND
FDA approves AMX0035 for the treatment of adults with MND

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