Rilutek (riluzole, by Sanofi) has been available in the U.S. as 50 mg tablets since December 1995. However, ALS patients can have difficulties with swallowing (dysphagia) due to muscle weakness or spasticity in the tongue, lips, palate, jaw, pharynx, larynx, and upper trunk.
Tiglutik is a thickened liquid formulation of riluzole (5 mg/mL), administered twice daily via an oral syringe, and is expected to be easier for patients to swallow. It was developed by ITF Pharma, a subsidiary of Italfarmaco.
“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 (Rilutek) as the gold standard of treatment,” Hiroshi Mitsumoto, MD, a 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 press release.
Mitsumoto also said that a liquid formulation of the treatment “may provide an opportunity for more accurate dosing and enhanced patient compliance.”
Added Calaneet Balas, The ALS Association’s president and CEO, “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.”
Denny Willson, ITF Pharma’s CEO, said the company expects to begin making Tiglutik available to ALS patients in the U.S. in mid-October.
While the treatment’s price is not yet known, Willson added that affordable access is a goal. “We have partnered with a specialty pharmacy to create a simple and straightforward product support program that will help patients receive Tiglutik quickly and with ease,” he said.
The FDA’s decision was based on studies of bioavailability – the proportion of a compound that circulates in the blood and able to have a therapeutic effect – comparing oral riluzole tablets to Tiglutik’s oral suspension.
The new formulation was approved under the FDA’s fast track designation, which speeds the review of potential treatments for serious conditions and that fill an unmet medical need. Tiglutik was also designated an orphan drug by the FDA.
According to ITF Pharma, the most frequent side effects of Tiglutik match those of riluzole, and include hypoesthesia (partial or total reduction of the sensitivity to stimuli), asthenia (lack of energy and strength), nausea, reduced lung function, hypertension, and abdominal pain.
For Tiglutik’s full prescribing information, please click here.
Although riluzole’s exact mechanism of action remains unclear, data from clinical trials has indicated that it inhibits both the release and signaling of the excitatory neurotransmitter glutamate, which may lessen neurodegeneration.