Mandos Health® is a biopharmaceutical company committed to furthering development of adrabetadex* for Niemann-Pick disease type C, an ultra-rare genetic disease that affects the body’s ability to metabolize cholesterol and lipids within cells. Niemann-Pick disease type C can affect the brain, nerves, liver, spleen, bone marrow and lungs.

The company is led by a diverse and passionate management team experienced in every stage of the drug development process from research through commercialization.

Our leadership team collectively has over 200 years of experience and has been intimately involved with 62+ drug approvals across 17 therapeutic areas, 5 CNS (central nervous system) and 7 rare and ultra-orphan disease drug or biologic therapies. We have executives that have worked in every stage of development from early research through commercialization.

Mandos has adequate supply to support the continuation of the EAP in North America. Importantly, Mandos will regularly update patients, families and advocates on our expanded access plans in North America and possibilities for countries outside North America.

We are exploring all possibilities to ensure access for any family affected by NPC. Please refer to the Expanded Access Policy. We will work diligently with regulators and scientific advisors and provide the community updates through our website.

Mandos is not currently focused on addressing media or press inquiries and is, instead, focusing on the Expanded Access Program, Investigator-Initiated Trials, and engaging with health authorities.

Our goal is to provide updates to the NPC community proactively through regular News on our website. Mandos believes in equitable News that deliver information to all community members at the same time.

Please see our Expanded Access Policy (EAP) page here.

We strive to be transparent with the community in everything we do to support a development plan for adrabetadex. Right now, we are exploring all options including re-evaluating existing data, exploring alternative trial design and endpoints with investigators and other members of the scientific and medical community. We aim to discuss our findings with regulators at the appropriate time and we will be expeditious in sharing progress updates via our website.

Mandos is now the official Sponsor and owner of all development programs involving adrabetadex since its acquistion of adrabetadex from Mallinckrodt. Mandos has taken on responsibility for all development programs for adrabetadex.

We continue to support access for families affected by NPC, with an immediate focus on those patients currently receiving treatment who wish to continue in the Expanded Access Program.