Shares of Orphazyme A\/S (ORPH) soared as much as over 1,300%, touching an intraday price of $77.77, yesterday, as the company awaits FDA word on its lead drug candidate MIPLYFFA.<\/p>\n
MIPLYFFA, known generically as Arimoclomol, is proposed for the treatment of Niemann-Pick type C, a rare inherited lysosomal storage disorder, with the regulatory agency’s decision expected on June 17, 2021.<\/p>\n
If approved, MIPLYFFA would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) and the drug could achieve peak global sales of $340 million, according to analysts.<\/p>\n
Orphazyme acquired the rights to Arimoclomol from CytRx Corp. (CYTR) in 2011 by paying a one-time, upfront payment of $150,000. CytRx is also entitled to receive up to a total of $120 million in milestone payments upon the achievement of certain pre-specified regulatory and business<\/span> milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from Arimoclomol.<\/p>\n We had alerted readers to ORPH on May 5, 2021 when it was trading around $9. (Report titled “Orphazyme Betting Big On Arimoclomol”)<\/p>\n The stock closed yesterday’s trading at $21.00, up 302.30%.<\/p>\n FDA decision is a binary event as it is linked to volatility and uncertainty. <\/p>\n Will the FDA approve Orphazyme’s MIPLYFFA for the Niemann-Pick disease Type C indication or will it be sent back to the drawing board? Stay tuned for the update.<\/p>\n Related Reading<\/p>\n Orphazyme Betting Big On Arimoclomol<\/p>\n (https:\/\/www.rttnews.com\/Products\/BiotechInvestor\/story.aspx?id=3192181&type=bimb) <\/p>\n