Orphazyme A/S (NASDAQ: ORPH) has revealed Monday that it has submitted Marketing Authorisation Application (MAA) for Arimoclomol to European Medicines Agency (EMA). Arimoclomol is the investigational candidate of Orphazyme for the treatment of Niemann-Pick disease Type C (NPC).
Marketing Authorisation Application is an application lodged by the companies to gain the approval of authorities to bring a particular medicinal product in the market. FDA is currently involved in the review of Orphazyme’s arimoclomol.
If the company receives approval for Arimoclomol then it will be a major breakthrough for Orphazyme as the company is striving to gain the approval of Arimoclomol in major markets.
Furthermore, Arimoclomol has received the Orphan Drug Designation in the US and Europe. It has also received the Breakthrough Therapy Designations as well as FDA Fast Track Designation for Niemann-Pick disease Type C (NPC) in the US. Additionally, Orphazyme has also been awarded Rare Pediatric Disease Designations in the US for Arimoclomol.
Shares of Orphazyme A/S (NASDAQ: ORPH) went down 0.73% at $9.49 during the trading session of Friday. The company had a trading volume of 17.81K as compared to the average volume of 194.46K. In the past 52-weeks of trading, Orphazyme’s stock has changed between the 52-weeks low range of $8.89 and a high range of $11.07.
Furthermore, it has moved up 6.75% from its 52-weeks low and moved up -14.27% from its 52-weeks high. This company has a total market capitalization of $329.28 million at the time of writing.
Niemann-Pick Type C is a rare fatal neurovisceral disease. In this progressive disease person’s body is unable to transport Cholesterol and other fatty substances inside of the cell. NPC basically belongs to the family known as lysosomal storage diseases. Orphazyme has created the investigational drug Arimoclomol which improves the function of lysosomes in the body.