- The stock price of Orphazyme (NASDAQ: ORPH) increased by over 20% pre-market. These are the details.
The stock price of Orphazyme (NASDAQ: ORPH) increased by over 20% pre-market. The company stock price has been highly volatile over the past week.
On June 10, the company stock price increased by over 1,386% during intraday trading due to speculation over an FDA decision involving Arimoclomol. Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). And HSPs are able to rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes.
Arimoclomol — which is administered orally — has been studied in ten phase 1, four phase 2, and three pivotal phase 2/3 trials and it is in clinical development for NPC and Gaucher disease. And Arimoclomol has received orphan drug designation (ODD) for NPC in the US and EU.
Plus Arimoclomol has received fast-track designation (FTD) from the U.S. Food and Drug Administration (FDA) for NPC. Arimoclomol has also received breakthrough therapy designation (BTD) and rare-pediatric disease designation (RPDD) from the FDA for NPC.
Orphazyme’s applications for arimoclomol (to be branded MIPLYFFATM)1 for Niemann-Pick disease type C (NPC) are under priority review with the U.S. Food and Drug Administration with an expected PDUFA action date of June 17, 2021, as well as with the European Medicines Agency, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected later this year.
The company stock price fell by over 55.57% during the last trading session. That fall in the stock price was driven by the company issuing a statement saying it is unaware of any material change in its clinical development programs, financial condition, or results of operations that would explain such price volatility or trading volume that has occurred since June 10, 2021.
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