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Orphazyme A/S was a late-stage biopharmaceutical company headquartered in Denmark, focused on developing and commercializing novel therapies for severe, debilitating rare diseases. Its primary efforts centered on arimoclomol, a heat shock protein amplifier, investigated for conditions like Niemann-Pick disease Type C (NPC), Amyotrophic Lateral Sclerosis (ALS), and Inclusion Body Myositis (IBM). Despite promising early data, arimoclomol faced regulatory hurdles with both the FDA and EMA in 2021 and early 2022, leading to significant restructuring of Orphazyme. In May 2022, substantially all of Orphazyme's assets, including arimoclomol, were acquired by KemPharm (now Zevra Therapeutics), which is continuing its development.
Served as the central hub for Orphazyme's research and development, clinical operations, corporate strategy, and administrative functions during its operational years.
The facility likely comprised modern office spaces and potentially laboratory facilities equipped for biopharmaceutical research, typical for companies in the Medicon Valley cluster.
The work culture at Orphazyme was likely driven by a strong scientific focus, a mission-oriented approach to addressing rare diseases, and a collaborative environment essential for drug development.
This headquarters was the nerve center for Orphazyme's global efforts to develop and seek approval for arimoclomol, coordinating international clinical trials and regulatory submissions.
Historically, Orphazyme's global presence was primarily characterized by its extensive clinical trial programs for arimoclomol, which were conducted across numerous sites in North America and Europe. The company actively engaged with international regulatory authorities, including the U.S. FDA and the European Medicines Agency (EMA), and collaborated with a global network of researchers, medical institutions, and patient organizations focused on rare neurodegenerative diseases.
Ole Maaløes Vej 3
Copenhagen
Capital Region of Denmark
Denmark
Address: Historically maintained a U.S. subsidiary office (Orphazyme US, Inc.). Specific street address not consistently publicized.
To facilitate U.S.-based clinical development for arimoclomol, navigate the complex FDA regulatory landscape, and prepare for potential future commercialization in the significant U.S. market.
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As of April 2025, Orphazyme' leadership includes:
Orphazyme has been backed by several prominent investors over the years, including:
Orphazyme experienced significant leadership transitions in early 2022, notably a CEO change, as the company navigated critical financial and regulatory challenges leading up to its restructuring and the sale of its assets. Many roles were impacted by widespread layoffs during this period.
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Orphazyme historically likely used common corporate email naming conventions. However, following the company's restructuring and the sale of its assets (including the orphazyme.com domain, which now redirects to zevra.com), email addresses associated with @orphazyme.com are no longer active or monitored.
Common patterns likely included [firstinitial][lastname]@orphazyme.com or [first].[lastname]@orphazyme.com
Format
example: jdoe@orphazyme.com or john.doe@orphazyme.com (These are illustrative examples and are no longer functional)
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Fierce Biotech / Company Announcements • May 17, 2022
After entering in-court restructuring proceedings in Denmark due to financial distress and regulatory setbacks, Orphazyme A/S announced the sale of substantially all its assets, including its lead candidate arimoclomol, to KemPharm (which subsequently rebranded as Zevra Therapeutics). The deal involved an upfront payment and potential future milestone payments, marking the effective end of Orphazyme's independent operations....more
Orphazyme Press Release / Endpoints News • February 23, 2022
Orphazyme announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion recommending against marketing authorization for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC). This followed an earlier rejection by the FDA, compounding the company's challenges....more
Orphazyme Press Release / BioSpace • June 18, 2021
Orphazyme A/S received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its New Drug Application (NDA) for arimoclomol for Niemann-Pick disease type C. The FDA stated that additional data were needed to support the application, a major setback for the company and its lead drug candidate....more
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