International Niemann–Pick Disease Alliance

Expanded Access Programme (EAP) – An Update from Orphazyme

Today, the INPDA received the attached letter (copied below), from Regan Sherman, Associate Director, Patient Advocacy Relations at Orphazyme.  The letter provides the latest update regarding the introduction of an EAP programme for their investigational therapy, arimoclomol.  Please do share this update with your communities as you feel appropriate.

If you have any questions, please contact Regan direct, using the details below, at any time.

Dear Members of the INPDA,

As many of you know, since mid-2019 our team at Orphazyme has been in discussions with regulatory agencies and local authorities regarding our intent to file a New Drug Application with the Federal Drug Administration (FDA) in the United States (US) and a Marketing Authorization Application with the European Medicines Agency (EMA) to seek approval of arimoclomol as a treatment for NPC.  As we progress towards filing, we have also been working diligently towards making arimoclomol available to NPC patients pre-commercially through an early access program (EAP). In many countries, EAP provides a pathway for patients with serious, life-threatening diseases or conditions who lack therapeutic alternatives to gain access to investigational drugs before they are approved.

Despite our best efforts, I regret to share with you that we will not meet our goal of introducing an EAP this fall. However, I can assure that development of the program is very much under way and we look forward to announcing its availability in early 2020.

Orphazyme’s primary aim is to obtain approval for arimoclomol as a treatment for NPC in the US and EU, with the long-term goal of achieving access for all patients in need. To ensure we maintain the focus and resources to achieve these goals, early access to arimoclomol as an investigational therapy must necessarily be made available on a country-by-country rolling basis, factoring in our progress towards regulatory filings, approval and local laws governing access. For these reasons, Orphazyme’s EAP will plans to initially provide access to arimoclomol pre-commercially to individuals with NPC in the United States.

Orphazyme has been engaged with the global NPC community for a number of years and we remain committed to doing what we can for NPC patients in need. And yet, despite our encouraging clinical development progress in NPC to date, the reality is that we are a pre-commercial stage company with lean resources conducting clinical development programs spanning four rare diseases. As a company that does not yet generate revenue, at this time we are not in a position to offer access to arimoclomol free of charge to all people living with NPC. It is our intent to offer early access to a number of additional countries over time, contingent upon discussions with local authorities and our progress towards filing for regulatory approval or reimbursement.

Please know that we fully appreciate the urgent need to bring arimoclomol to patients with NPC and embrace our responsibility to achieve this goal. I have attached a letter from our Chief Medical Officer, Thomas Blaettler, MD, which you are welcome to share more broadly with your constituents as you feel appropriate. I am also happy to speak with any of you directly regarding questions; however, we are not able to comment on specific country timelines or progress towards availability of EAP outside of the US at this time.






Regan Sherman

Associate Director, Patient Advocacy Relations

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