Rilutek (riluzole)

The first drug ever approved for ALS and still the most widely prescribed. Riluzole is thought to work by reducing the toxic effects of glutamate on motor neurons. In trials it modestly lengthened the time to tracheostomy or death (median survival was about 2 to 3 months longer), but measures of muscle strength and nerve function did not show a benefit.

In ALS, motor neurons are damaged by too much glutamate, a chemical that normally helps nerve cells communicate. Riluzole is thought to reduce the amount of glutamate released by nerve cells, although exactly how it helps in ALS is not known. By calming this chemical overload, it may protect remaining motor neurons from further damage and help people live longer.

Common side effects include weakness, nausea, dizziness, decreased lung function, and stomach (abdominal) pain. Liver enzyme levels went above normal in about half of patients in the main trials, so blood tests to check the liver are needed before starting and during treatment. Less common effects include vomiting, dry mouth, and trouble sleeping. Serious risks on the label include liver injury and pancreatitis (both of which can be fatal), lung inflammation (interstitial lung disease), and a severe drop in infection-fighting white blood cells (neutropenia), which has been reported in the first 2 months of treatment. Tell your doctor right away if you have a fever, a dry cough or trouble breathing, stomach pain, nausea, vomiting, loss of appetite, yellow skin or eyes, or dark urine.

Taken as an oral tablet, 50 mg twice daily. Should be taken at least one hour before or 2 hours after meals. Also available as an oral suspension (Tiglutik) for patients who have difficulty swallowing. The Exservan oral film has been discontinued in the US.

Yes, Riluzole (Rilutek) is FDA approved (1995) for the treatment of Amyotrophic Lateral Sclerosis.

Riluzole extends median survival by approximately 2 to 3 months and improves the probability of surviving one year by about 9%, according to Cochrane meta-analyses of multiple clinical trials. While this may sound modest, it represents a meaningful benefit for many patients, particularly when combined with other treatments and supportive care. Riluzole modestly extends survival but does not stop or reverse the disease.

Yes. In the US, riluzole is available as generic oral tablets and as an oral liquid suspension (Tiglutik) for patients who have difficulty swallowing tablets. The Rilutek brand tablet and the Exservan oral film have been discontinued in the US. Both available forms deliver the same active ingredient. The liquid form is especially helpful as ALS progresses and swallowing becomes more difficult, and it can also be given through a feeding (PEG) tube.

Liver function tests (ALT, AST) should be checked before starting riluzole and during treatment, and the label advises watching for liver injury every month for the first 3 months and from time to time after that, since liver enzyme rises are common and serious liver injury can happen. Tell your doctor about any fever, because riluzole can cause a severe drop in white blood cells (neutropenia). Most liver enzyme elevations are mild and reversible, but treatment should be stopped if levels rise significantly. Your neurologist will set a monitoring schedule based on your individual risk factors.

Yes. Riluzole is commonly used alongside edaravone, and patients in the tofersen trials were also taking riluzole. There are no major drug interactions that prevent combination therapy with currently approved ALS treatments. Riluzole works through a different mechanism (glutamate reduction) than other ALS drugs, making it complementary rather than redundant.

Yes. Generic riluzole tablets contain the same active ingredient at the same dose and must meet FDA bioequivalence standards. Generic versions have been available since 2013 and are significantly less expensive than branded alternatives. The brand-name Tiglutik suspension offers dosing convenience for patients with swallowing difficulties but are not more effective than generic tablets for patients who can swallow pills.

Riluzole is approved and recommended for all forms of ALS regardless of genetic subtype, including both sporadic and familial cases. Its mechanism of reducing glutamate toxicity addresses a pathway common to all ALS, not a specific genetic mutation. It is typically started at diagnosis and continued throughout the disease course as part of standard care.

Riluzole is generally continued for as long as the patient tolerates it, throughout the entire disease course. There is no established stopping point. If side effects become problematic, your neurologist may adjust the dose or discuss alternatives, but the default approach is long-term continuous treatment.