• Orphazyme supports consensus guidelines to direct interactions with rare disease patient advocacy organizations

  • Company extends heartfelt gratitude and respect to patient organization partners during Niemann-Pick Disease Awareness Month


Copenhagen, October 15, 2018 – Orphazyme A/S, a biopharmaceutical company dedicated to developing treatments for patients living with rare diseases, today announced the company’s endorsement of published guidelines to support interactions with rare disease patient advocacy organizations.

Published in the Orphanet Journal of Rare Diseases earlier this year, Principles for interactions with biopharmaceutical companies: The development of guidelines for patient advocacy organizations in the field of rare diseases, is a landmark article for the rare disease community. Toni Mathieson, Chief Executive of Niemann-Pick UK (NPUK), an organization that works in partnership with Orphazyme, is a co-author on the paper.

Anders Hinsby, Chief Executive Officer of Orphazyme, said: “Close collaboration with patient organizations is essential to bringing new therapies to people living with rare diseases. We have been privileged to work with Niemann-Pick disease patient organizations, such as NPUK, for nearly a decade. The knowledge and experience shared by these groups have been instrumental in guiding our path.”

Anders added, “The standards and recommendations put forth in this paper will further guide our interactions with non-profit organizations to ensure transparent, ethical partnerships as we increase our engagement with the communities representing Inclusion Body Myositis (IBM), Amyotrophic Lateral Sclerosis (ALS), and Gaucher disease.

Paul Merrigan, Chief Commercial Officer and President of Orphazyme US, said: “People living with rare disorders and their families are the authorities on these diseases; we rely on the expertise of patient organization partners to guide our actions. For these reasons, our first US employee was hired to create a patient advocacy relations program, built on the foundation of Orphazyme’s longstanding commitment to collaborating with patient communities.”

Orphazyme colleagues who oversee engagement with patient communities will attend the National Organization of Rare Diseases (NORD) Rare Summit this week in Washington, D.C. We welcome the opportunity to meet with patient organizations working to support NPC, ALS, IBM, and Gaucher disease communities.