The U.S. Food and Drug Administration has agreed to expand Tiglutik, a liquid oral form of riluzole, to include giving it via a feeding tube to treat amyotrophic lateral sclerosis (ALS), ITF Pharma, the company marketing Tiglutik, announced.
The approval of administering the therapy via percutaneous endoscopic gastrostomy (PEG) — a tube placed through the abdominal wall into the stomach — makes Tiglutik available for more patients, including those who require the use of a feeding tube because they have difficulty swallowing.
“We are very pleased the FDA has approved the application to broaden the existing label for Tiglutik to include PEG tube administration. This news marks another important step forward in the treatment of ALS and addresses an unmet treatment need of the ALS community,” Peter Cook, president and CEO of ITF Pharma, said in a press release.
Riluzole has been available in the U.S. in tablet form (brand name Rilutek, by Sanofi) since December 1995. But ALS patients often have difficulties swallowing, a consequence of muscle weakness, and the administration of tablets can be difficult.
Tiglutik is a thickened liquid formulation of riluzole, approved in 2018 as the first and only easy-to-swallow oral suspension of riluzole for the treatment of ALS. It was designed to be administered with an oral syringe, which is more comfortable for patients.
Having an oral suspension formulation precludes the need for patients or caregivers to handle tablets, easing administration and perhaps providing an opportunity for more accurate dosing and better patient compliance.
However, the formulation was not available for patients requiring a feeding tube to maintain their nutritional and hydration status.
“There are many medical and quality of life advantages for the use of a PEG feeding tube as an individual’s ALS progresses, including the ability maintain more adequate hydration, administer medications and to potentially reduce the risk of choking and aspirating when oral ingestion becomes too challenging,” said Benjamin Rix Brooks, MD, medical director at the Carolinas Neuromuscular/ALS – MDA Center.
The recent approval was based on results from an open-label crossover study with healthy volunteers, where an into-the-stomach administration of Tiglutik was equivalent in safety and pharmacokinetics to receiving it orally. (Pharmacokinetics is the study of how medicines are absorbed, distributed, metabolized, and eliminated from the body.)
The study included 32 participants, average age 37.3 years, who received a single dose of Tiglutik orally or via a feeding tube. Then, after at least five days of a washout period, participants were transposed to the other treatment arm.
Both methods of administration were safe and well-tolerated, and showed similar a pharmacokinetics profile. The most common side effects were in agreement with previous studies, and included a reduced sense of sensation in the mouth (for oral route only), physical weakness, nausea, decreased lung function, high blood pressure, and abdominal pain.
“This expanded use for Tiglutik provides an alternate administration route for the majority of people with ALS who choose to undergo this procedure,” Brooks said.