ITF Secures FDA Approval for Liquid Riluzole for ALS Patients

Privately-held ITF Pharma snagged regulatory approval for Tiglutik, a liquid formulation of riluzole for the treatment of amyotrophic lateral sclerosis.

Pennsylvania-based ITF, a subsidiary of Italfarmaco, said the approval makes Tiglutik the “first and only easy-to-swallow thickened riluzole liquid” for patients with ALS.  The liquid formulation of riluzole will make it easier for ALS patients to take the medication due to a difficulty in swallowing. The medication was approved under the U.S. Food and Drug Administration (FDA)’s fast-track designation, which speeds up the approval process when there is an unmet need.

Riluzole is designed to delay the onset of a dependence of a ventilator in ALS patients. It was initially approved by the FDA in 1995.

Hiroshi Mitsumoto, a professor of neurology at Columbia University at The Neurological Institute of New York, called the approval of the liquid formulation a “welcome step forward” for treating ALS patients. He called riluzole the “gold standard” for slowing the progression of ALS in patients over the past 20 years.

“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,” Mitsumoto said in a statement.

Denny Willson, chief executive officer of ITF Pharma, said the company plans to have the liquid formulation available for patients by the middle of October through its “highly-specialized field sales team.” Willson added that the company is committed to helping ALS patients have affordable access to Tiglutik. He said the company partnered with a specialty pharmacy that will “create a simple and straightforward product support program” to help patients receive the treatment in as easy a manner as possible.

“This reflects our underlying mission to provide valuable therapeutic options and support programs that make a positive difference in the lives of both patients and healthcare providers,” Willson said in a statement.

ALS (amyotrophic lateral sclerosis) is a progressive neurodegenerative disease that affects neurons in the brain and the spinal cord. The disease is usually fatal within two to five years of diagnosis. Patients with ALS eventually lose the ability to control muscle movement, which eventually leads to total paralysis and then death. Currently, there is no known cure for the disease. The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 diagnosed annually. 

While Riluzole has been a standard of treatment for ALS, it’s not the only option on the block of prescribers. Last year Mitsubishi Tanabe and its new subsidiary, MT Pharma America, secured regulatory approval for Radicava, the first ALS drug approved in 22 years. Clinical data demonstrated patients who received Radicava for six months experienced significantly less decline in physical function — by 33 percent.

Other companies are also in the middle of developing ALS treatments, including Israel-based BrainStorm. That company’s NurOwn uses a patient's own cells which have been engineered outside the body, to produce and secrete factors known to promote neuronal survival.