Newron Pharmaceuticals Receives FDA Orphan Drug Designation For Sarizotan For The Treatment Of Rett Syndrome

MILAN--(BUSINESS WIRE)--Newron Pharmaceuticals S.p.A. (“Newron”), a research and development company focused on novel central nervous system (CNS) and pain therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Newron’s New Chemical Entity (NCE) sarizotan for treatment of Rett syndrome. Newron also recently announced that it received a positive opinion from the Committee for Orphan Medicinal Products (COMP) from the European Medicines Agency (EMA) for sarizotan.

“Newron believes that sarizotan could demonstrate an improvement in key symptoms of Rett syndrome, including episodes of apnea, hyperventilation and breath-holds”

“Newron believes that sarizotan could demonstrate an improvement in key symptoms of Rett syndrome, including episodes of apnea, hyperventilation and breath-holds,” said Ravi Anand, Chief Medical Officer at Newron. “A reduction in respiratory symptoms is likely to improve quality of life of patients, caregivers, and in the long term, we expect that treatment with sarizotan may reduce secondary cardio-respiratory complications and extend the lives of girls and women with Rett syndrome. Newron is currently in advanced discussions with regulatory authorities in Europe, the United States and Canada on the proposed clinical development program.”

Rett syndrome is a severe neurodevelopmental disorder primarily affecting females with an estimated prevalence ranging from one in 10,000 to 20,000 females. There are no approved treatments available. Rett syndrome is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction which leads to loss of ability to conduct daily life activities, walk or communicate. Rett syndrome also is associated with a reduced life expectancy. Approximately 25 percent of the deaths in patients with Rett syndrome are possibly related to multiple cardio-respiratory dysrhythmias that result from brain stem immaturity and autonomic failure. More than 95 percent of these patients have a mutation in the MeCP2 gene. Prevalence of episodes of apnea, hyperventilation and disordered breathing are found in approximately 70 percent of patients with Rett syndrome at some stage of their life.

Sarizotan, a 5HT1A agonist and D2 agonist/antagonist, has been associated with a 70 to 85 percent reduction of apneas and hyperventilation episodes in preclinical testing with both acute and chronic dosing. Sarizotan has been fully characterized in preclinical studies evaluating its toxicological effects and metabolic profile, without any significant safety concerns.

About the Orphan Drug Designation

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act (ODA), including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee.

About Newron Pharmaceuticals

Newron (SIX: NWRN) is a biopharmaceutical company focused on the development of novel therapies for patients with diseases of the central nervous system (CNS) and pain. The Company is headquartered in Bresso near Milan, Italy. Marketing authorization in the EU for Xadago® (safinamide) was granted by the EU Commission in February 2015, followed by the launch in the first key EU country - Germany - in May 2015. The New Drug Application (NDA) to the U.S. FDA has been accepted for filing as reported in March 2015. In March 2014, Zambon, a partner of Newron, submitted a MAA to Swissmedic. Zambon has the rights to develop and commercialize safinamide globally, excluding Japan and other key Asian territories where Meiji Seika has the rights to develop and commercialize the compound. Newron’s additional projects are based on highly promising treatments for rare disease patients and are at various stages of clinical development, including sarizotan for patients with Rett syndrome, sNN0031 for patients with Parkinson’s disease, non-responsive to oral drug treatments, sNN0029 for patients with ALS and ralfinamide for patients with specific rare pain indications. Newron is also developing NW-3509 as the potential first add-on therapy for the treatment of patients with positive symptoms of schizophrenia.

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