FDA Clears Liquid Riluzole (Tiglutik) for ALS

Megan Brooks

September 06, 2018

The US Food and Drug Administration (FDA) has approved a liquid form of riluzole (Tiglutik, ITF Pharma) for patients with amyotrophic lateral sclerosis (ALS). Tiglutik is administered twice daily via an oral syringe.

"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, from the Neurological Institute of New York and New York-Presbyterian Hospital/Columbia University Medical Center, New York City, said in a news release issued by the company.

"The availability of Tiglutik oral suspension precludes the need for manipulation of tablets by patients or caregivers, easing administration and may provide an opportunity for more accurate dosing and enhanced patient compliance," said Mitsumoto.

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

Results of bioavailability studies comparing riluzole tablets to oral suspension riluzole led to approval of the liquid form.

The side effect profile of oral suspension riluzole mirrors that of riluzole tablets, the company said, and include oral hypoesthesia, asthenia, nausea, decreased lung function, hypertension, and abdominal pain.

"While riluzole's mechanism of action is not fully understood, in clinical studies it has been shown repeatedly to modulate glutamate neurotransmission by inhibiting both glutamate release and postsynaptic glutamate receptor signaling," the company said.

Oral suspension riluzole had FDA fast-track and orphan drug designations.

ALS, also known as Lou Gehrig's disease, is a progressive, ultimately fatal neurodegenerative disease, marked by a gradual degeneration of nerve cells of the central nervous system that control voluntary muscle movement.  An estimated 5000 people in the United States are diagnosed with ALS each year. It is estimated that more than 20,000 Americans have the disease at any given time.

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