Orphazyme

About Orphazyme

Orphazyme ApS operates as a biopharmaceutical company focused on developing and commercializing therapies for rare, neurodegenerative diseases. The company was established in 2009 by co-founders Thomas Kirkegaard Jensen and Marja Jäättelä. The foundation of Orphazyme stems from research into heat shock proteins (HSPs) conducted at the Danish Cancer Society, where the founders identified their potential in treating protein misfolding diseases.

The company's primary drug candidate was arimoclomol, an orally administered small molecule designed to amplify the production of heat shock proteins. This mechanism aimed to rescue misfolded proteins, clear protein aggregates, and improve lysosomal function, targeting the underlying pathology of several neurodegenerative conditions. Orphazyme's pipeline primarily targeted diseases such as Niemann-Pick disease type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and sporadic Inclusion Body Myositis (sIBM). The business model centered on obtaining regulatory approval for arimoclomol and subsequently commercializing it for these specific orphan disease markets, which are characterized by high unmet medical needs. Revenue generation was contingent on future drug sales following successful clinical trials and market authorization.

Orphazyme achieved several significant milestones, including listing on the Nasdaq Copenhagen in 2017 and later on the Nasdaq U.S. However, the company faced major setbacks. In 2021, the U.S. Food and Drug Administration (FDA) issued a Complete Response Letter (CRL), declining to approve the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C. Subsequently, in 2022, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) also adopted a negative opinion, recommending the refusal of the marketing authorization for arimoclomol in the European Union. These regulatory failures precipitated a severe financial crisis for the company, leading to a delisting from Nasdaq U.S. and an in-court restructuring process in Denmark, which ultimately resulted in the company filing for bankruptcy in March 2022. In a significant development following the bankruptcy, KemPharm acquired substantially all of Orphazyme's assets, including arimoclomol, with the intention of further developing the drug candidate.