Approved Treatments for ALS

Exservan (riluzole oral film) uses Aquestive Therapeutics’ PharmFilm technology. It’s an orange film containing a 50 mg riluzole dose that is taken twice a day by placing it on the tongue. It dissolves automatically and requires no water. The active ingredient in Exservan is also available as oral tablets (Rilutek) and as an oral suspension (Tiglutik).

Qalsody (tofersen) is an injection therapy conditionally approved in the U.S. for the treatment of ALS associated with mutations in the SOD1 gene (SOD1-ALS). The therapy has been shown in clinical trials to significantly slow disease progression and extend survival, but the approval was based on biomarker data that the regulatory agency said is reasonably likely to predict a clinical benefit in patients.

Radicava (edaravone) is the first treatment for ALS that the U.S. Food and Drug Administration approved in more than 20 years. The therapy is designed to slow the decline of physical function in people with ALS. The therapy also has been approved in Canada, where it is available under a number of different medication plans.

Relyvrio (sodium phenylbutyrate and taurursodiol) was an approved oral treatment for adults with ALS that has now been withdrawn from markets in the U.S. and Canada. Early clinical trials showed its ability to slow functional declines and to extend survival, but no benefits were observed in a confirmatory Phase 3 trial.

Rilutek (riluzole) was the first treatment approved by the U.S. Food and Drug Administration to treat ALS in 1995. It is an oral formulation that acts to slow the progression of ALS symptoms and prolong survival. The exact mechanism by which Rilutek treats ALS is unknown. However, it is thought that the medicine blocks the release of glutamate from nerve cells.

Tiglutik (riluzole oral suspension) was approved by the U.S. Food and Drug Administration in September 2018. It is a liquid oral formulation of riluzole. Because ALS patients have difficulties swallowing, the administration of tablets can be difficult. Tiglutik is administered with an oral dosing syringe, which is more comfortable for patients.

There are also a number of therapies available that can help to treat the symptoms of ALS. As the disease can progress differently in each individual patient, the symptomatic therapies a patient is prescribed should be tailored to their needs. These therapies can help treat muscle symptoms, excessive saliva production, pseudobulbar affect, among a range of other issues. Learn more about some of these therapies at the links below.