- The stock price of Orphazyme A S (NASDAQ: ORPH) increased by over 80% today. This is why it happened.
The stock price of Orphazyme A S (NASDAQ: ORPH) increased by over 80% today. This is a continuation of momentum as the company stock price has spiked multiple times as the scheduled date for an FDA approval approaches.
Last week I wrote about how the stock price of ORPH increased 1,386% as well. Investors are rallying around a decision from the FDA involving arimoclomol. Arimoclomol is an investigational drug candidate that amplifies the production of heat shock proteins (HSPs). 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 10 phase 1, 4 phase 2, and 3 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. However, it has not been approved by the FDA yet.
It is now under the FDA priority review with a PDUFA action date scheduled for tomorrow.
Disclaimer: This content is intended for informational purposes. Before making any investment, you should do your own analysis.
