The US Food and Drug Administration (FDA) has granted approval for Amylyx Pharmaceuticals’ Relyvrio (sodium phenylbutyrate and taurursodiol) to treat adult patients with amyotrophic lateral sclerosis (ALS).
Indicated for use as a monotherapy or along with currently approved therapies, Relyvrio can be administered orally by mixing a packet in eight ounces of water.
For the first three weeks, the recommended dose is one packet a day. Following this time period, the dosage is raised to one packet twice daily.
The FDA approval is based on findings from the multicentre Phase II CENTAUR clinical trial that enrolled 137 ALS patients.
The study also had a randomised, placebo-controlled phase of six months as well as an open-label extension (OLE) long-term follow-up phase.
According to the findings, the trial met its primary efficacy endpoint.
Additionally, treatment with Relyvrio substantially reduced physical function loss in ALS patients.
Diarrhoea, nausea, upper respiratory tract infection and abdominal pain were reported to be the most common adverse events linked to Relyvrio.
An oral, fixed-dose medicine, Relyvrio is also approved with conditions in Canada such as Albrioza to treat ALS.
The Marketing Authorisation Application submitted by the company for the therapy is being reviewed by the European Medicines Agency (EMA) for ALS.
Amylyx Pharmaceuticals Commercial global head and chief commercial officer Margaret Olinger said: “Our priority now is to ensure that adults living with ALS in the US whose doctors have prescribed Relyvrio can access it as quickly as possible.
“Physicians will be able to prescribe immediately, and we anticipate speciality pharmacies will be able to start to fill prescriptions and ship Relyvrio to people with ALS in the next four to six weeks.”
A progressive and fatal neurodegenerative ailment, ALS is caused by the death of motor neurons in the brain and spinal cord.