RARE Daily

Orphazyme Lays Off Two-Thirds of Staff After FDA Setback

June 29, 2021

Orphazyme announced a restructuring that included a two-thirds reduction in staff following the recent refusal of the U.S. Food and Drug Administration to approve arimoclomol, the company’s experimental therapy for the rare lysosomal storage disorder Niemann-Pick disease type C.

Photo: Christophe Bourdon, Orphazyme CEO

The company said the restructuring is intended to enable it to advance its corporate strategy and the development of arimoclomol for Niemann-Pick disease type C. The company said it is committed to pursuing regulatory approval in Europe and assessing a path forward for arimoclomol in the United States.

“As a result of the restructuring of the company and our rigorous cost saving program, we will have to part ways with many of our most valued and talented colleagues,” said Christophe Bourdon, Orphazyme CEO. “The immediate actions we are taking are necessary to protect and support the ongoing approval process in Europe and the evaluation of a path forward in the U.S.”

As part of the restructuring, Orphazyme said it will globally reduce the number of employees to those who will support essential activities moving forward. In Denmark, Orphazyme will immediately initiate negotiations under the Danish Act on Collective redundancies and the Act on Information and Consultation.

As part of the restructuring, Rémi Droller, Martijn Kleijwegt, and Anders Hedegaard will resign from the Orphazyme board of directors effective June 30, 2021.

Niemann-Pick disease type C (NPC) is a genetic, progressively debilitating, fatal neurovisceral disease. As a consequence of a genetic mutation, patients with the condition produce a misfolded NPC protein that prevents the body from clearing certain lipids. As these lipids build-up in tissues and organs, including the brain, it causes progressive damage. The estimated prevalence of NPC in the United States and Europe combined is 1,000-2,000. There are no approved treatments for NPC in the United States and only one approved product in Europe.

Arimoclomol is an experimental drug that amplifies the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol has received Fast-Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation from the FDA for NPC. On June 17, 2021, Orphazyme received a Complete Response Letter from the FDA regarding its New Drug Application for arimoclomol for the treatment of NPC.

The FDA told the company it needed qualitative and quantitative evidence to substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale (NPCCSS) and, in particular, the swallow domain. A primary endpoint of the phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. This is a disease-specific measure of disease progression consisting of the five clinically most relevant domains to patients with NPC, caregivers, and physicians.

Author: Rare Daily Staff

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