International Niemann–Pick Disease Alliance

Updates

  1. Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency

    DOWNLOAD THE PDF VERSION Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency Xenpozyme represents first Sanofi therapy to be approved under the SAKIGAKE ‘fast-track’ designation Approval based on positive results from two separate clinical trials...

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  2. Orphazyme Update – March 2022

    We are writing to inform you that the CT-ORZY-NPC-002 trial activities as well as ongoing early-access and named-patient programs for arimoclomol in NPC will continue for the current time. As you may have seen, Orphazyme participated in an ad-hoc...

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  3. Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

    – Second Orphan Drug Designation Supports Azafaros’ Strategy to Develop AZ-3102 as a Disease Modifying Treatment in a Range of Severe Rare Inherited Metabolic Disorders – Leiden, The Netherlands, March 24, 2022 – Azafaros B.V. today announced that the...

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  4. Orphazyme announces withdrawal of European Marketing Authorisation Application for arimoclomol for the treatment of Niemann-Pick disease type C

    March 22, 2022 06:54 ET | Source: ORPHAZYME A/S Orphazyme A/S Company announcement No. 13/2022 Inside information www.orphazyme.com Company Registration No. 32266355 Decision to withdraw the European Marketing Authorisation Application comes ahead of scheduled final vote on the application later this month Orphazyme intends to request Type C Meeting with...

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  5. The Global Community supports INPDA’s letter to EMA

    The International Niemann-Pick Disease Alliance (INPDA) has organised a letter (see below) voicing the perspective and concerns of the NPC community, which will be sent to the European Medicines Agency (EMA) next week. Families also have the opportunity to...

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  6. Orphazyme Announces Update on Regulatory Review of Arimoclomol in the European Union

    Orphazyme A/S Company announcement No. 07/2022 Inside information www.orphazyme.com Company Registration No. 32266355 Copenhagen, Denmark, February 23, 2022 – Orphazyme A/S (ORPHA.CO (DK); ORPH (US)) (the “Company”), a late-stage biopharmaceutical company, today announced an update on the ongoing review of the Marketing Authorisation Application (MAA)...

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  7. 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 an update from IntraBio, regarding the clinical development of their lead drug, N-acetyl-L-leucine (IB1001) for NPC.  IntraBio is conducting a pivotal Phase III, randomized, double-blind, placebo-controlled trial for patients aged 4 years and older...

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  8. Orphazyme provides update for planned NDA resubmission for arimoclomol for the treatment of Niemann-Pick disease type C in the United States

    Orphazyme A/S Investor news No. 02/2022 www.orphazyme.com Company Registration No. 32266355 Orphazyme has made progress towards resubmission of the NDA for arimoclomol to the FDA and plans to request a Type C Meeting in Q2 2022 Subject to these discussions, the Company aims...

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  9. Cyclo Therapeutics Announces Formation of Global Steering Committee Comprised of Leading Experts to Advise on the Global Phase 3 Clinical Development Program for Trappsol® Cyclo™ in Niemann-Pick Disease Type C

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  10. Changes to the Executive Management of Orphazyme – Anders Vadsholt to be appointed CEO

    Copenhagen, Denmark, January 31, 2022 – Orphazyme A/S (ORPHA.CO; ORPH) (“Orphazyme” or the “Company”), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, today announces that Chief Executive Officer (CEO) Christophe Bourdon...

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