What are the upcoming developments in the Amyotrophic Lateral Sclerosis (ALS) Treatment Landscape?

What is Amyotrophic Lateral Sclerosis?[besut | besut sumber]

Amyotrophic Lateral Sclerosis (ALS) is a nervous system disease that weakens muscles and impacts physical function.

The treatment landscape for amyotrophic lateral sclerosis is rigorous and comprises multidisciplinary care, including physical therapy, speech therapy, food counseling, heat or whirlpool therapy, and other practices. The following medications have been licensed for the treatment of amyotrophic lateral sclerosis: RILUZOLE, RADICAVA, TIGLUTIK, EXSERVAN, and RELYVRIO. There is currently no viable treatment to slow or stop the disease's progression from ALS. Palliative care and symptom management are the cornerstones of ALS treatment.

Riluzole and its formulations approval journey[besut | besut sumber]

The FDA authorized riluzole in 1995 as the first medication for the treatment of ALS. Many other nations recommended the medication as the first-line therapy for the treatment of ALS throughout the next decades. It is advised to take riluzole to increase survival rates and/or time till tracheostomy. The tablet form of the medication has restrictions that may have limited its clinical use. Poor oral bioavailability, detrimental effects of food on the body, particularly the liver, pharmacokinetic unpredictability, and oral numbness are just a few of the medication's major drawbacks. There are generic forms of riluzole, which has been used as an ALS medication in the United States for more than 20 years. According to secondary findings, riluzole is used more than 80% of the time in the 7MM.

RADICAVA, the first Amyotrophic Lateral Sclerosis medication in decades since Riluzole approval with an eye-popping price tag[besut | besut sumber]

The second medication used to treat ALS patients is called RADICAVA (edaravone), and it slows the decline in physical function in ALS patients. It took nearly 20 years for RADICAVA to receive approval for treating ALS, despite the fact that it has been used to treat acute stroke in Japan since 2001 under the brand name RADICUT. Based on a series of ALS clinical trials conducted in Japan, it was licensed for the treatment of acute stroke in Japan in June 2015 and in South Korea in December 2015.

After learning that edaravone was being used to treat ALS in Japan, the US FDA was so delighted with Mitsubishi's medication that it contacted Mitsubishi right away to discuss submitting a marketing application in the US.

Application withdrawal of RADICAVA from EU after CHMP requested a new trial[besut | besut sumber]

The primary research did reveal a substantial improvement in ALSFRS-R scores in patients receiving RADICAVA compared to those getting a placebo, according to the Committee for Medicinal Products for Human Use (CHMP). The trial only included a small number of patients, and there was insufficient proof that other crucial metrics, like those pertaining to survival, respiration, and muscle strength, had improved. There was no discernible difference when participants in the placebo group were later transferred to RADICAVA.

What do we know about this new drug, RELYVRIO?[besut | besut sumber]

The FDA authorized Amylyx Pharmaceuticals' AMX0035, a co formulation of sodium phenylbutyrate and taurursodiol, for the treatment of ALS in September 2022. The medication, RELYVRIO, was approved as the third therapy to assist decrease ALS disease progression or mortality. Additionally, the company's marketing authorization application for AMX0035 for the treatment of ALS in Europe is being evaluated by the European Medicines Agency (EMA).

What is expected from the ALS treatment market in the future?[besut | besut sumber]

In general, licensed medications like RADICAVA, RELYVRIO, and other riluzole formulations make up the market for ALS treatments today. NUEDEXTA, on the other hand, just treats symptoms; it has no impact on slowing the progression of the illness. RNS60 (RevalesioCorporation), EPI-589 (PTC Therapeutics), AT-1501 (Eledon Pharmaceuticals), AP-101 (AL-S Pharma), ANX005 (Annexon), Pegcetacoplan (Apellis Pharmaceuticals), PrimeC(NeuroSense Therapeutics), and BIIB078 are just a few of the promising mid- and early-phase treatments in the ALS pipeline (Biogen). Although there are a number of promising treatments for amyotrophic lateral sclerosis in various phases of research, there have been numerous ups and downs and failed clinical trials throughout the history of ALS drug development.

The problem is the fact that the patients do not have enough time to survive after they get diagnosed with the disease.

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