International Niemann–Pick Disease Alliance

Updates

  1. IB1001-301 Recruitment Complete

    IntraBio Ltd. is pleased to confirm that it has completed recruitment for its Pivotal Trial, Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study (IB1001-301 – NCT05163288). Enrolment for IB1001-301 commenced...

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  2. Cyclo Therapeutics Announces Publication of Phase 1 Data for Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1 (NPC1)

    – Data published in official journal of the Society for Inherited Metabolic Disorders, Molecular Genetics and Metabolism – Published trial data show that Trappsol® Cyclo™ overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the...

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  3. Xenpozyme (olipudase alfa) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations)

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  4. NNPDF GOES VIRTUAL – NNPDF Family Support & Medical Conference 2022

    Want to join the NNPDF Family Support & Medical Conference but can’t be there in person? VIRTUAL Conference registration is available! Cost is per family (includes multiple devices) and includes main sessions. Check out the agenda at t.ly/agenda2022 for all LiveStream...

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  5. Press Release: Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD

    Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD Paris, June 28, 2022. The European Commission (EC) has approved Xenpozyme® (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of...

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  6. IntraBio: IB1001-301 (International Recruitment)

    IntraBio Inc. Pivotal Trial Announcement: IB1001-301, “Effects of N-Acetyl-L-Leucine on Niemann-Pick disease type C (NPC): A Phase III, randomized, placebo-controlled, double-blind, crossover study.” We are pleased to share the news that recruitment to IntraBio’s Pivotal trial: IB1001-301 “Effects of...

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  7. Mandos NPC1 Community Newsletter, May 2022

    May 2022 To The NPC1 Community, Happy May. It’s been a little while since we provided our last communication back in February and we’re excited to share three important updates on our progress. We have completed our first meeting...

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  8. TransportNPC: Scott Fine, CEO of Cyclo Therapeutics

    Dear Members of the NPC Community, My name is Scott Fine and I have been CEO of Cyclo Therapeutics since 2015. Our company is enrolling a Phase 3 study of a potential treatment for NPC. The study, called “TransportNPC”,...

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  9. CHMP recommends approval of Xenpozyme® (olipudase alfa), the first and only treatment for ASMD

     Recommendation based on positive results from two clinical trials in which Xenpozyme provided improvement across multiple non-CNS clinical manifestations of ASMD in pediatric and adult patients  ASMD is a rare, progressive, and potentially life-threatening disease with no...

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  10. Orphazyme A/S under In-Court-Restructuring to sell substantially all of its assets and business activities to KemPharm, Inc.

    Orphazyme A/S in restructuring Company announcement No. 24/2022 Inside information www.orphazyme.com Company Registration No. 32266355 KemPharm to acquire Orphazyme assets, including those relating to the development and approval of arimoclomol, for a total of USD 12.8 million in cash and...

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