Israel is poised to become the first country outside North America to use a new ALS drug, which is believed to extend patients’ lives by almost a year on average.
Neopharm, headquartered in Petach Tikva, has signed a distribution deal with Massachusetts-based Amylyx Pharmaceuticals to distribute AMX0035 — marketed as Relyvrio — in Israel, the West Bank and Gaza.
The drug is today only available in the US and Canada, and an Amylyx statement announcing the Neopharm deal, released on Tuesday, said it could prove to be an “important step” towards roll-out in the wider Middle East, Europe and Africa.
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. It leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually death.
Clinical data based on a trial suggested that AMX0035 slowed the progression of the disease. Amylyx reported that in trials it increased survival by a median of 10.6 months. An expert unconnected to Amylyx and Neopharm, Yale University neurologist Dr. Babar Khokhar, wrote in a briefing that the trial data translates to a slowing in decline of about 25%.
When AMX0035 was approved by America’s Food and Drug Administration in September it became just one of three ALS medications green-lighted for use.
The others are Rilutek, which was approved in 1995, and Radicava, approved in 2017. AMX0035 can be taken with other ALS medicines, and many doctors are hoping that it complements their benefits.
“There is tremendous excitement in the field,” Dr. Michael Abrahamy, medical director of Neopharm, told The Times of Israel on Thursday. “The ALS community is in great need of treatments. The disease quickly takes people from being independent to wheelchair-bound, and then with high mortality. The prospect of slowing progression is exciting.”
AMX0035, which is given orally, is intended to stop neurons from dying and therefore to slow ALS progression.
The clinical data which led to FDA approval indicated that the medication slowed the progression of the disease. Neopharm needs to secure the approval of Israel’s Ministry of Health before starting local distribution, but it is very rare for a drug approved by the FDA to be rejected in Israel.
When the FDA gave its approval in September, the drug had only completed a 137-participant Phase II trial, not the larger Phase III trial that it normally requires.
Khokhar wrote in his briefing: “Most drugs go through the usual process of clinical trial Phases I, II, and III; if there’s good efficacy and safety data, then they are approved. But ALS is so rare, and there is no treatment to stop the disease or significantly reverse it, so the FDA said they felt compelled to approve it.”
Abrahamy said that he believes the medicine offers hope in the face of a fast-progressing illness. “It’s a disease which, in the space of three-and-a-half years from diagnosis, can commonly take people from a healthy life to deterioration,” he commented. “Patients are always looking for hope and it’s great that Israel will become the third country in the world to benefit from a new source of hope.”