The fourth "gradually frozen" drug! US FDA approved exservan to treat als

November 27, 2019 / BIOON / -- aquestive therapeutics is a professional pharmaceutical company dedicated to using innovative technology to develop differentiated products to solve treatment challenges for patients The company is promoting a pipeline of late patent products for the treatment of central nervous system diseases and providing alternatives to invasive standard care therapies In addition, the company works with other pharmaceutical companies to bring new molecules to market using its proprietary, best of breed technologies, such as pharmfilm Recently, the company announced that exservan, an oral film developed by the company, was approved by the US FDA one week ahead of schedule for the treatment of ALS patients Exservan has been granted orphan drug qualification (odd) by FDA Orphan drugs are drugs used to prevent, treat and diagnose rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases In the United States, rare diseases refer to the types of diseases with a population of less than 200000 Incentives for drug research and development of rare diseases include various incentives for clinical development, such as tax credits related to clinical trial costs, FDA user fee relief, FDA assistance in clinical trial design, and a seven-year market monopoly period after the drug is approved for marketing Amyotrophic lateral sclerosis (ALS) is an irreversible fatal motor neuron disease The main symptoms are progressive muscle weakness and atrophy of limbs and trunk muscles, and gradually lose motor function, like being "frozen", so it is commonly known as "frozen man" The disease generally develops rapidly, more than half of the patients have an average survival time of 3-5 years after diagnosis, and most of them die of respiratory failure caused by respiratory myasthenia It is estimated that the incidence rate of ALS worldwide is about 2/100000 Although about 5% - 10% of cases are related to heredity, the etiology of ALS is still unknown, which may involve genetic and environmental factors At present, the drugs on the market can only delay the progress of the disease, and there is no drug that can cure ALS Exservan is a riluzole, the first FDA approved drug to treat ALS The tablet product was launched in 1995 to delay ventilator dependence So far, four drugs have been approved by FDA to treat ALS, three of which are different dosage forms of riluzole products: exservan (oral film agent of riluzole), Rilutek (tablet of riluzole), tiglutik (suspension of riluzole), radicava (edaravone, intravenous injection solution of Edaravone) Among the four drugs mentioned above, radicava from Mitsubishi Taguchi was approved by FDA in 2017, which is the first ALS new drug approved by the agency in the past 22 years Edaravone (Edaravone), the active drug component of radicava, is a kind of free radical scavenger, which is believed to alleviate the effects of oxidative stress, which may be the key factor in the pathogenesis and development of ALS The antioxidant effect of edaravone is believed to provide neuroprotective support for the nervous system, potentially delaying disease development or limiting additional damage In China, radicava was approved by the National Drug Administration (nmpa) at the end of July It is worth noting that it took less than two months for the drug to get priority review and approval It is estimated that up to 30000 people in China are affected by ALS Original source: aquestive therapeutics receipts FDA approval for exservan Gamma (riluzole) Oral Film