ALS drug pulled from market in US, Canada after trial shows it doesn’t work

The pharmaceutical company behind a drug for amyotrophic lateral sclerosis (ALS) said it would start a process to remove it from the US and Canadian markets.

Amylyx Pharmaceuticals said the drug for ALS also known as motor neurone disease (MND) or Lou Gehrig’s disease would no longer be available for new patients after a trial showed it did not work.

“Patients currently on therapy in the US and Canada who, in consultation with their physician, wish to stay on treatment can be transitioned to a free drug [programme],” the company said.

Trials showed no difference to placebo

Earlier this month, Amylyx said that a trial’s results involving 664 participants showed there was no significant difference between participants taking the drug, marketed as Relyvrio or Albrioza, compared to those taking a placebo.

MND is an uncommon but eventually fatal disease affecting people’s ability to control their arms and legs. It can also lead to trouble breathing.

While there is no cure for the disease, some treatments can reduce its impact, according to the UK’s National Health Service (NHS).

There are no changes for the drug in Europe, as the European Medicines Agency (EMA) refused a marketing authorisation for the drug last year.

“At the time of the initial opinion in June 2023, the Agency had concerns that the main study did not show convincingly that Albrioza was effective in slowing down the worsening of the disease,” the EMA said.

“Data on survival were also not reliable, given the way the data were collected and analysed,” it added.

The US Food and Drug Administration (FDA), however, approved the drug in 2022.

Amylyx said on Thursday it will lay off 70 per cent of its more than 350 employees as part of a major restructuring effort.

Company executives said they plan to continue studying Relyvrio and another experimental drug for several rare diseases, including Wolfram syndrome, which causes childhood diabetes and blindness.

‘Regulatory flexibility’

The 2022 approval was mainly based on results from one small, mid-stage study that was criticised by some of the agency’s own internal scientists.

Normally the agency requires two large, late-stage studies that show a clear benefit before granting approval.

But at the time FDA officials explained that “regulatory flexibility” was appropriate when reviewing Relyvrio, “given the serious and life-threatening nature of ALS and the substantial unmet need”.

The medication is part of a string of drugs for deadly, degenerative diseases that have won FDA approval in recent years despite questionable evidence they work.

Relyvrio is a powder that combines two older drugs: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine.

Amylyx faced criticism for pricing the drug at $158,000 (€146,251) for a year’s supply. Sales were disappointing, with some patients discontinuing the medicine after only a few months.