Amyotrophic lateral sclerosis (ALS) is a condition which affects the bodies nerve cells. It’s neurodegenerative and progressive, causing the patient muscle weakness and muscle atrophy. It eventually results in paralysis. 80% of people with the condition have trouble swallowing, as it takes more than 26 muscles to do so. Unfortunately, the only approved treatment for the disease requires swallowing.
But hopefully it won’t be that way for long.
The drug is called riluzole. So far, it is the only drug to extend tracheostomy-free survival that has been approved by the FDA. It currently comes as a pill or as a thick liquid (oral suspension), both methods that require swallowing. The drug has been previously granted Orphan Drug designation by the FDA.
Biohaven has just created a way to administer the treatment that simply requires putting a tablet underneath your tongue. From there it dissolves sublingually, eliminating the swallowing issue for patients. In clinical trials, patients found the drug easy to use.
It’s called BHV-0223 and it’s a lower dose of riluzole (40mg) than the pill or oral suspension (50mg), but the study showed similar blood exposures of the drug in patients with all administering methods. If approved, BHV-0223 will be the only available effective treatment for ALS which doesn’t require swallowing.
Biohaven has submitted their application for review, and the application has been accepted. This is an extremely hopeful first step toward FDA approval.
Stay tuned for future updates on this development! If approved, it could be life-changing for ALS patients.
You can read more about this development here.