Roquette, a leading provider of pharmaceutical excipients and global leader in plant-based ingredients, is pleased to announce a strategic investment with Beren Therapeutics P.B.C., and the launch of an innovation agreement to expand the full potential of Beren’s cyclodextrin technologies and their medicinal applications.

We have secured new long-term funding that lets us commit to this work for the long haul. What matters most to us is what that makes possible. We are building a set of programs designed to support families across the full NPC journey, from the first search for a diagnosis through the day-to-day of care, whatever the road ahead holds.

During the webinar, Jason Camm, Chief Executive Officer, Beren Therapeutics P.B.C., shared an important milestone.

We are pleased to share that on December 17, 2025, Beren Therapeutics P.B.C.®, through its subsidiary Mandos LLC®, submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for adrabetadex. This application seeks approval for the treatment of patients with infantile-onset Niemann-Pick disease type C (NPC).

Our entire team of over 60 dedicated professionals at Mandos Health® is thrilled to share a new update as we continue our commitment to transparency, collaboration, and progress in Niemann-Pick disease type C (NPC) for the global NPC community.

We are excited to share important updates on our progress as we continue to build a science-driven organization dedicated to NPC.

Hello and Happy 2025! We are excited to share important updates on our progress as we continue to build a science-driven organization dedicated to NPC.

We are excited to share important updates on our progress and commitment to supporting novel therapies for Niemann-Pick Type C (NPC) Disease.

We are excited to share important updates on our progress and commitment to supporting novel therapies for Niemann-Pick Type C (NPC) Disease.

We are excited to share important updates on our progress and commitment to supporting the Niemann-Pick Type C (NPC) Disease.

We are excited to share important updates on our progress and commitment to supporting the Niemann-Pick Type C (NPC) Disease.