Community Update — Recognizing Niemann-Pick Awareness Month

Dear NPC Community,

For families living with NPC, awareness matters when it changes what happens next: an earlier diagnosis, better support, or more time with a child.

Beren was founded on the belief that our responsibility does not end with developing medicines. We should identify and remove every barrier that stands between a patient and  achieving the best possible long-term outcome.

This belief shapes how we are building Beren and our commitment to the NPC community.

Finding children earlier. We recently launched NPC GenomeComplete with GeneDx because too many children with NPC remain undiagnosed for too long. The program provides eligible families with no-cost comprehensive genome sequencing, family testing and genetic counseling. Our goal is simple: make it easier to identify NPC earlier and shorten the path families travel before getting an answer.

You can help us reach children with NPC earlier. Please share NPC GenomeComplete with the healthcare providers, families and others in your networks who may encounter a child still searching for a diagnosis. Healthcare providers and families can learn more about eligibility and ordering at TestforNPC.com.

Giving families a permanent seat at the table. If we are going to understand and remove the barriers to better outcomes, families need a permanent voice in how we make decisions. The NPC Community Council brings caregivers and advocates directly into our thinking about diagnosis, access, family support and long-term care. It makes permanent something we have believed since Beren's inception as a Public Benefit Corporation: the lived experience of families should help shape how we serve the community.

Preparing for what comes next. Adrabetadex remains an investigational medicine, and the FDA is reviewing our New Drug Application for infantile-onset NPC, with a target action date of November 17, 2026.  

Across Beren, we are preparing for the potential approval of adrabetadex so that, following an FDA decision, we are ready to make it available to eligible patients as quickly as possible. Our Expanded Access Program continues throughout the review and remains available to eligible patients.

These initiatives are different, but they come from the same founding belief: Our responsibility begins before a medicine is approved and continues long after. It means diagnosing children earlier, understanding and removing the barriers to access, and continuing to invest in what can improve outcomes for children and their families over the long term.

During NPC Awareness Month, you will also find our team at:  

  • the Child Neurology Society (CNS) Annual Meeting (October 14–17, Montreal, Canada)
  • the American Society of Human Genetics (ASHG) Annual Meeting (October 20–24, Montreal, Canada)
  • the BIO Patient Advocacy Changemakers Event (October 22–23, Washington, D.C.)
  • the NORD Rare Diseases & Orphan Products Breakthrough Summit (October 25–27, Washington, D.C.)

If you are attending, we would be pleased to connect.

Thank you for continuing to place your trust in us and for helping shape the company we are building.

With gratitude,

Jason

Chief Executive Officer
Beren Therapeutics, P.B.C.

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