The U.S. Food and Drug Administration will allow MonoSol Rx to use a new form of its Riluzole therapy, Riluzole Oral Soluble Film (Riluzole OSF), to treat amyotrophic lateral sclerosis (ALS) before the agency approves the new therapy.
The FDA granted MonoSol Rx’s request that Riluzole OSF receive Investigational New Drug status, which means it can be used to help patients with limited approved treatment options and unmet medical needs.
The designation will allow MonoSol Rx to start clinical trials of Riluzole OSF.
The previous version of Riluzole, which blocks the amino acid glutamine that is used in protein formation, is the only FDA-approved ALS therapy. It can delay patients going on a ventilator or having a tracheostomy — surgery to create an airway in the windpipe — and may increase their survival for two to three months.
It is not a cure for ALS, however, and it does not reverse the nerve damage or muscle weakness the disease causes. A number of tests have proven it safe.
The original developer of Riluzole was Rhone-Poulenc Rorer, which later became known as Sanofi-Aventis. The therapy is available as a tablet under the trade name Rilutek or as a liquid under the trade name Teglutik. The liquid formulation may be more suitable for patients with swallowing difficulties.
Researchers have also studied the treatment’s ability to address mood and anxiety disorders and Alzheimer’s disease.
ALS kills neurons that control muscles. Its manifestations include stiff muscles, muscle twitching, and gradually worsening weakness due to muscles decreasing in size. The condition leads to difficulty speaking, swallowing, and eventually breathing.
About 6,000 a year develop the disease in the United States, according to the ALS Association.
“ALS is a highly debilitating neurodegenerative disease that has a tremendous impact on patients’ and caregivers’ lives,” Keith Kendall, CEO of MonoSol Rx, said in a press release. The company looks forward “to initiating our pivotal pharmacokinetic clinical study for Riluzole OSF,” he said.