FAQs

Mandos is a biopharmaceutical company committed to furthering development of adrabetadex* for Niemann-Pick Type C, an ultra-rare genetic disease that affects the body’s ability to metabolize cholesterol and lipids within cells. Niemann-Pick Type C disease can affect the brain, nerves, liver, spleen, bone marrow and lungs and there are currently no FDA approved treatments. 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 is working with Mallinckrodt to secure sufficient supply to support the continuation of the EAP in North America beyond October 20th, 2021. Importantly, Mandos will regularly update patients, families and advocates on our expanded access plans in North America and rest of world.
We are exploring all possibilities to ensure access for any family affected by NPC1. Specifically, we are looking into both EAP and Investigator-Initiated Trials (IITs), reanalyzing the existing clinical data, and actively working in parallel to engage with the FDA on the EAP and a potential development path.

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 securing additional supply of adrabetadex.

Our goal is to provide updates to the NPC community proactively through regular communications on our website. Mandos believes in equitable communications that deliver information to all community members at the same time.
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.
Our interactions with Mallinckrodt to date have been collaborative and focused on a shared vision of developing adrabetadex for patients with NPC1.

Mandos is now the official Sponsor of all clinical programs involving adrabetadex and has partnered with Mallinckrodt to continue the wind-down of the existing clinical trials (VTS301, VTS-270-001, and VTS-270-302) while Mandos takes on responsibility for all other clinical programs and future development efforts.

We consider Mallinckrodt an active part of the NPC community and we look forward to working closely with them.
We are exploring all possibilities to support access for families affected by NPC1, with an immediate focus on those patients currently receiving treatment who wish to continue.

For more details of our Expanded Access Policy, please click here.