- Orphazyme A/S (NASDAQ:ORPH) plans an approximate two-thirds reduction in its global workforce due to a restructuring, aiming to advance its corporate strategy and the development of arimoclomol for Niemann-Pick disease type C (NPC).
- The company further said it remains committed to pursuing regulatory approval in Europe and assessing a path forward for arimoclomol in the U.S. after being hit by a Complete Response Letter from the FDA last week.
- NPC is a rare, genetic, progressively debilitating, and often fatal neurovisceral disease.
- Regarding its trading, Orphazyme said its financial outlook remains unchanged for 2021.
- With the planned restructuring and resulting cost savings, the company intends to free resources.
- Concurrently, the company announced 24-month interim results of an open-label extension trial for arimoclomol in NPC.
- The data were shared at the Parseghian Scientific Conference for Niemann-Pick disease type C Research.
- A slowing of progression from baseline was observed through 36 months in participants who received arimoclomol from the start of the double-blind phase (mean change, 3.5 points).
- The effect was consistent across pre-specified subgroups, including those treated with miglustat among participants more than four years of age.
- Also, slowing of progression through 24 months was observed in those participants who initiated arimoclomol treatment upon entering the open-label period (mean change, 0.9 points).
- Price Action: ORPH shares are down 1.78% at $7.71 during the premarket session on the last check Monday.
