Clinigen, Mitsubishi link for early access programme

ALS is an idiopathic neurodegenerative disease in which motor neurons selectively degenerate and vanish, affecting around two in 100,000 people worldwide.

The cause for the majority of cases is not well understood, but may involve genetic and environmental factors.

Edaravone, which was discovered by Mitsubishi, is approved for use as a treatment for ALS in Japan, South Korea and the US.

While it is yet to receive approval in Europe, the groups’ early access programme aims to get some patients treated with the drug outside of the clinical trial setting.

Edaravone scavenges free radicals, which increase in ALS, protecting motor neurons from oxidative stress and delaying the decline in muscle strength and the progress of muscular atrophy.

In clinical trials, people given edaravone showed significantly less decline - by 33 percent - in physical function compared to placebo as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).

"We are pleased to once again be working with MTPC to help eligible patients in Europe gain access to this important medicine," noted John Lagus, Head of Managed Access, Clinigen.

"As the trusted global leader in access to unlicensed medicines, this partnership aligns with our mission of getting the right medicines to the right patient at the right time."

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