What Does Orphazyme Do?

Total employees85
HeadquartersCopenhagen
Founded2009

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.

Where Is Orphazyme's Headquarters?

HQ Function

Served as the central hub for Orphazyme's research and development, clinical operations, corporate strategy, and administrative functions during its operational years.

Notable Features:

The facility likely comprised modern office spaces and potentially laboratory facilities equipped for biopharmaceutical research, typical for companies in the Medicon Valley cluster.

Work Culture:

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.

HQ Significance:

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.

Values Reflected in HQ: The headquarters aimed to embody Orphazyme's commitment to scientific innovation, patient-centricity in rare diseases, and perseverance in tackling complex medical challenges.

Location:

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.

Street Address:

Ole Maaløes Vej 3

City:

Copenhagen

State/Province:

Capital Region of Denmark

Country:

Denmark

Where Else Does Orphazyme Operate Around the World?

Chicago, Illinois, USA

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.

Buying Intent Signals for Orphazyme

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Notable news
Hiring actively
Corporate Finance
Corporate Finance
Corporate Finance
Corporate Finance
Corporate Finance
*Example signal, not calculated in real time

Who's Leading Orphazyme? Meet the Executive Team

As of April 2025, Orphazyme' leadership includes:

Patrik Dahlen - Former Chief Executive Officer (during 2022 restructuring)
Anders Vadsholt - Former Chief Financial Officer
Christophe Bourdon - Former Chief Executive Officer (until March 2022)
Thomas Blaettler - Former Chief Medical Officer
Anders Hinsby - Former Chief Executive Officer (until March 2021)

Who's Investing in Orphazyme?

Orphazyme has been backed by several prominent investors over the years, including:

Novo Holdings A/S
Sunstone Life Science Ventures
Aescap Venture
Kurma Partners
Idinvest Partners
Coöperatieve LSP U.A. (LSP)
Various public and institutional shareholders (post-IPO)

What Leadership Changes Has Orphazyme Seen Recently?

Hire1
Exits1

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.

Departures

Christophe Bourdon, Christophe Bourdon resigned as CEO amidst the company's challenges.

New Appointments:

Patrik Dahlen, Patrik Dahlen was appointed as the new CEO to lead Orphazyme during its critical restructuring phase.

What Technology (Tech Stack) Is Used byOrphazyme?

Discover the tools Orphazyme uses. Highperformr reveals the technologies powering your target accounts — helping your sales, marketing, and GTM teams prioritize smarter and close faster.

Orphazyme Email Formats and Examples

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)

Example

0%

Success rate

What's the Latest News About Orphazyme?

Fierce Biotech / Company AnnouncementsMay 17, 2022

Orphazyme Sells Key Asset Arimoclomol to KemPharm (now Zevra Therapeutics) Following Restructuring

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 NewsFebruary 23, 2022

Orphazyme Receives Negative Opinion from EMA for Arimoclomol in Niemann-Pick Disease Type C

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 / BioSpaceJune 18, 2021

Orphazyme Receives Complete Response Letter from FDA for Arimoclomol in Niemann-Pick Disease Type C

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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