The approval was based on bioavailability studies which compared the tablet formulation of riluzole to the oral suspension formulation.
Hiroshi Mitsumoto, MD, DSc
The FDA has granted approval to ITF Pharma for its riluzole oral suspension (Tiglutick) for the treatment of amyotrophic lateral sclerosis (ALS).
It was previously granted Fast Track and Orphan Drug designations from the agency. The approval marks the first and only easy-to-swallow thickened riluzole liquid for ALS, administered twice daily through an oral syringe. It is the seventh approval of the therapy worldwide.
"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, said in a statement.1
Mitsumoto, the 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, noted that the availability of the oral suspension halts the need for patients or caregivers to manipulate oral tablets, making administration easier and possibly providing an opportunity for more accurate dosing and improved patient adherence to treatment regimens.
The approval from the FDA was based on bioavailability studies which compared the tablet formulation of riluzole to the oral suspension formulation.2 Study data showed that the total exposure of riluzole 50-mg tablets and the riluzole 5 mg/mL oral suspension was essentially equivalent (ratio, 106.84%; 90% CI, 96.98 to 117.71). The oral suspension resulted in a more rapid absorption (Tmax, ~30 mins) with a maximum concentration that was roughly 20% higher than the tablets (ratio, 122.32%; 90% CI, 103.28 to 144.88).
Study authors noted that as the suspension requires no manipulation or premixing, it represents an advantageous method of delivery, with little to no anesthetic effect in the mouth, as more than 80% of patients with ALS may become unable to swallow solid oral doses.
The most common adverse events (AEs) were oral hypoesthesia, asthenia, nausea, decreased lung function, hypertension and abdominal pain, which were consistent with the known safety profile of riluzole.
Calaneet Balas, MBA, MSc, the president and chief executive officer of the ALS Association, called the approval and “important step forward in the treatment of ALS,” in a statement. While the mechanism of action for the therapy remains to be completely understood, clinical trials have shown that it modulates glutamate neurotransmission by inhibiting both the release of glutamate and postsynaptic glutamate receptor signaling.
"We are very pleased with the FDA approval of TIGLUTIK and we look forward to making the first and only easy-to-swallow thickened riluzole liquid for ALS commercially available in the U.S. in mid-October through our highly-specialized field sales team," said Denny Willson, the chief executive officer of ITF Pharma, in a statement. "ITF is committed to supporting the ALS community and to helping people living with ALS find affordable access to TIGLUTIK. Therefore, 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. 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."
1. ITF Pharma Announces FDA Approval Of TIGLUTIK™ (Riluzole) Oral Suspension for The Treatment Of Amyotrophic Lateral Sclerosis (ALS) [press release]. Berwyn, PA: ITF Pharma; Published September 6, 2018. prnewswire.com/news-releases/itf-pharma-announces-fda-approval-of-tiglutik-riluzole-oral-suspension-for-the-treatment-of-amyotrophic-lateral-sclerosis-als-300707653.html. Accessed September 6, 2018.
2. Dyer AM, Smith A. Riluzole 5 mg/mL oral suspension: for optimized drug delivery in amyotrophic lateral sclerosis. Drug Des Devel Ther. 2017:11:59-64. doi: 10.2147/DDDT.S123776.