Niemann-Pick Type C Pipeline Outlook Report 2025: Key 10+ Companies and Breakthrough Therapies Shaping the Future Landscape

DelveInsight's, "Niemann Pick C Disease Pipeline Insight, 2025" report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in Niemann Pick C Disease pipeline landscape. It covers the Niemann-Pick Type C pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Niemann-Pick Type C pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Niemann-Pick Type C Pipeline Report

* In July 2025, Azafaros A.G. announced a Phase 3 study that will evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in multiple disease areas using a Master Protocol Research Program. Participants are randomized to different subprotocols based on disease type: Niemann-Pick type C (NPC), GM1 gangliosidosis or GM2 gangliosidosis.
* DelveInsight's Niemann-Pick Type C pipeline report depicts a robust space with 10+ active players working to develop 15+ pipeline therapies for Niemann-Pick Type C treatment.
* The leading Niemann-Pick Type C Companies such as Zevra Therapeutics, Inc, Azafaros, Cyclo Therapeutics, IntraBio, SOM Biotech, ENDECE and others.
* Promising Niemann-Pick Type C Pipeline Therapies such as arimoclomol, IB1001, Miglustat, Hydroxypropyl-beta-cyclodextrin, Nizubaglustat, miglustat and others.

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Niemann-Pick Type C Emerging Drugs Profile

* Arimoclomol: Zevra Therapeutics, Inc

Arimoclomol is an orally delivered, first-in-class investigational product candidate for the treatment of NPC, has been granted Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, and Rare Pediatric Disease designation by the FDA, and Orphan Medicinal Product designation for the treatment of NPC by the European Medicines Agency (EMA). In December 2023, Zevra Therapeutics, Inc announced it resubmitted its New Drug Application for arimoclomol to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days. Zevra expects the NDA to be classified as a Class II submission which would be subject to a review period by the FDA within six months from the date of submission. The FDA has accepted the resubmission of the NDA for arimoclomol and has set a user fee goal date (PDUFA date) of September 21, 2025. Currently the drug is been registered of development for the treatment of Niemann Pick C Disease.

* AZ 3102: Azafaros

AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C). In 2023, AZ-3102 received three Rare Pediatric Disease Designations (RPDD) from the United States Food and Drug Administration (FDA) for the treatment of GM1 and GM2 gangliosidoses, and NP-C. In 2022, the compound received Fast Track Designation for NPC and Orphan Drug Designations (ODD) for NP-C from the FDA. Currently the drug is in Phase II for the treatment of Niemann-Pick Disease Type C (NP-C).

The Niemann-Pick Type C Pipeline report provides insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Niemann-Pick Type C with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Niemann-Pick Type C Treatment.
* Niemann-Pick Type C Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Niemann-Pick Type C Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Niemann-Pick Type C market.

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Niemann-Pick Type C Companies

Zevra Therapeutics, Inc, Azafaros, Cyclo Therapeutics, IntraBio, SOM Biotech, ENDECE and others.

Niemann Pick C Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

* Intravenous
* Subcutaneous
* Oral
* Intramuscular

Niemann-Pick Type C Products have been categorized under various Molecule types such as

* Monoclonal antibody
* Small molecule
* Peptide

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Scope of the Niemann-Pick Type C Pipeline Report

* Coverage- Global
* Niemann-Pick Type C Companies- Zevra Therapeutics, Inc, Azafaros, Cyclo Therapeutics, IntraBio, SOM Biotech, ENDECE and others.
* Niemann-Pick Type C Pipeline Therapies- arimoclomol, IB1001, Miglustat, Hydroxypropyl-beta-cyclodextrin, Nizubaglustat, miglustat and others.
* Niemann-Pick Type C Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Niemann-Pick Type C Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Which companies are leading the race in Niemann-Pick Type C drug development? Find out in DelveInsight's exclusive Pipeline Report-access it now! @ Niemann-Pick Type C Emerging Drugs and Major Companies [https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]

Table of Contents

* Introduction
* Executive Summary
* Niemann-Pick Type C Disease: Overview
* Niemann-Pick Type C Disease Pipeline Therapeutics
* Niemann-Pick Type C Disease Pipeline Therapeutic Assessment
* Niemann Pick C Disease- DelveInsight's Analytical Perspective
* Late Stage Products (Registration)
* Arimoclomol: Zevra Therapeutics, Inc
* Mid Stage Products (Phase II)
* AZ 3102: Azafaros
* Early Stage Products (Phase I/II)
* Drug Name: Company Name
* Preclinical and Discovery Stage Products
* Drug Name: Company Name
* Inactive Products
* Niemann-Pick Type C Disease Key Companies
* Niemann-Pick Type C Disease Key Products
* Niemann-Pick Type C Disease- Unmet Needs
* Niemann-Pick Type C Disease- Market Drivers
* Niemann-Pick Type C Disease Market Barriers
* Niemann-Pick Type C Disease- Future Perspectives and Conclusion
* Niemann-Pick Type C Disease Analyst Views
* Niemann-Pick Type C Disease Key Companies
* Appendix

About Us

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