FDA Approves Riluzole Oral Suspension for the Treatment of Amyotrophic Lateral Sclerosis

The US Food and Drug Administration (FDA) has approved ITF Pharma's riluzole (TIGLUTIK) oral suspension for the treatment of amyotrophic lateral sclerosis (ALS). To be administered twice daily via an oral syringe, riluzole is the first and only easy-to-swallow thickened riluzole liquid for patients with ALS.

Oral suspension, according to ITF Pharma, will make administration of the treatment easier, and fulfill a therapeutic need for about 80% of patients with ALS who go on to develop dysphagia, a swallowing impairment.

"Having a therapeutic option designed to specifically overcome the challenges of disease-related dysphagia in ALS is a welcome step forward for the many doctors, caregivers and people living with ALS who have relied on riluzole as the gold standard of treatment for more than 20 years to slow the progression of this devastating disease," Hiroshi Mitsumoto, MD, DSc, Wesley J. Howe professor of neurology at Columbia University at The Neurological Institute of New York and New York-Presbyterian Hospital/Columbia University Medical Center, said in a recent statement.

Bioavailability studies comparing oral riluzole tablets with riluzole oral suspension served as the basis for the approval.

In a single-blind study conducted by Ann Margaret Dyer. PhD and founder of PharmaSci Consulting Limited, Nottingham, UK, and Alan Smith, PhD, assessed diluzole 5 mg/mL oral suspension: for optimized drug delivery in amyotrophic lateral sclerosis.

The novel suspension formulation of riluzole was found to enable more accurate dosing and consistent ongoing administration. In addition, enhanced patient compliance was observed since swallowing can become difficult for patients with ALS. According to the study, “An oral liquid formulation provides an important therapeutic option for patients with ALS, >80% of who may become unable to swallow solid oral dosage forms due to disease-related dysphagia.”

Oral hypoesthesia, asthenia, nausea, decreased lung function, hypertension, and abdominal pain are the most common side effects of riluzole oral suspension; these side effects are consistent with the established clinical profile of riluzole.

By inhibiting both glutamate release and postsynaptic glutamate receptor signaling, riluzole has been shown to modulate glutamate neurotransmission repeatedly in clinical studies, despite the fact that the treatment’s mechanism of action is not fully understood.

"This approval marks an important step forward in the treatment of ALS. The ALS Association would like to thank the FDA and ITF Pharma for working together to bring this important new formulation of riluzole to the ALS community," Calaneet Balas, president and chief executive officer at The ALS Association, added.

Riluzole oral suspension was approved under FDA fast-track designation. Previously, riluzole was granted an orphan drug designation by the FDA.