New FDA-Approved Drug for ALS: AMX0035

New FDA-Approved Drug for ALS: AMX0035

Commonly referred to as Lou Gehrig’s Disease, ALS or Amyotrophic Lateral Sclerosis is a terrifying disease. It is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. The worst part is that there is no cure for it. Most medications treat it moderately at best. Now, a new medication has hit the market. It is called AMX0035. This newly approved medication is making waves in the ALS community.

What Exaclty is ALS?

It is unknown why some people develop ALS, although sometimes it is inherited in about 5-10% of cases. Researches believe there is a connection between genetic and environmental factors, but nothing concrete has been established. It most commonly affects adults between 40 and 65 with more men experiencing this condition.

This neurodegenerative disease slowly atrophies muscles. Essentially, ALS affects control of the muscles needed to move, speak, eat and breathe. Unlike dementia and alzheimers, people typically do not lose their ability to process their thoughts, just their ability to move and eventually to breathe.

ALS patients are sometimes given devices to help assist with breathing. They may be given a bilevel positive airway pressure (BiPAP) device for nighttime breathing. They may also undergo a tracheostomy. This is a surgically created hole, centered in the trachea for use with a respirator. Other issues that occur include speech issues that can helped by using a speaking device, malnutrition, dehydration, pneumonia and the development of dementia.

The lifespan after an ALS diagnosis also varies. Most people live between 3-5 years after receiving the diagnosis. Others have been known to live over 10 years.

People report their first symptoms as muscle twitching, and weakness in a limb and potentially slurred speech. The overall symptoms of the full blown disease vary greatly from patient to patient. They can include but are not limited to:

  • Tripping and falling
  • Difficulty walking or performing normal daily activities
  • Weakness in legs, feet or ankles
  • Hand weakness
  • Slurred speech or trouble swallowing
  • Muscle cramps and twitching in arms, shoulders and tongue
  • Inappropriate crying, laughing or yawning
  • Cognitive and behavioral changes

Additionally, there is typically no pain in the early stages of ALS or throughout the end stages.

Why is This New Treatment Important?

Simply put, there are not a lot of treatment options when someone is diagnosed with ALS. The outcomes are devastating and poor. With the FDA approving AMX0035 (a co-formulation of sodium phenylbutyrate and taurursodiol), it marks only the 3rd medications available for treating patients with ALS. Edaravone has been approved since 2017 and Riluzole has been used since 1995.

The approval of AMX0035 or CENTAUR is nothing short of unconventional. The Phase 2 study for the new medication only consisted of 137 participating patients. It proved to slow the functional decline of the patients over 24 weeks, but secondary outcomes were not vastly different between the control group and those who received AMX0035. It does appear that longer and larger trials are warranted.

In March 2022, the FDA decided to not approve this medication. They believed the trials were not sufficient enough to allow this approval. They voted down the use of the drug in 6-4 decision. However, by September of 2022, the FDA did eventually approve of AMX0035 for use in ALS patients.

So why did the FDA have a change of heart in September? Although the medication trial for AMX0035 was not very conclusive, the ALS community is extremely limited on therapeutics and treatment options. Given their short lifespan (2-5 years after diagnosis) and with no known cure, AMX0035 still provides a meaningful step forward, even if it just adds a few months to the lifespan of someone living with ALS. The FDA advisory committee reversed their previous decisions and approved the drug in a 7-2 vote.

What Does the Drug Maker Have to Say?

The drug maker of AMX0035, Amylyx is excited about the future of their medication. However they understand the trial was very limited. They have given their word that if the drug proves to not perform well over the coming months and years, they will voluntarily withdraw the medication. Subsequent trials will occur and will be very telling of the future of AMX0035.

Final Thoughts on ALS and AMX0035

The future of ALS remains grim, but drug makers are working diligent to find new treatments and potentially a cure. Most medications are incredibly expensive as well, so it makes a lot of treatment options very difficult to obtain. The oversight on new medications is sometimes not as particular since the treatment is desperately needed as patients have a very short lifespan after their initial diagnosis. Companies are continually working hard to help and support the ALS community.