Niemann Pick C Disease Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies

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 C Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the 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 C Disease Pipeline Report

* In March 2025, Actelion announced a study is conducted in Chinese subjects aged 4 years and older with Niemann Pick Type C disease (NPC). Approximately 19 subjects will be enrolled in this study. The study will be conducted at 2 sites in China. Patients will be treated with miglustat for 12 months, efficacy and safety outcomes will be measured.
* In March 2025, Azafaros A.G. conducted a phase 2 is a 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C disease (NP-C) patients. After approval by the country health authorities, a double-blind extension period was proposed to the patients who complete the 12-week study.
* DelveInsight's Niemann Pick C Disease pipeline report depicts a robust space with 10+ active players working to develop 15+ pipeline therapies for Niemann Pick C Disease treatment.
* The leading Niemann Pick C Disease Companies such as Zevra Therapeutics, Inc., Azafaros, Cyclo therapeutics, IntraBio, SOM Biotech, ENDECE and others.
* Promising Niemann Pick C Disease Pipeline Therapies such as Arimoclomol, N-Acetyl-L-Leucine, IB1001, Miglustat, Hydroxypropyl-beta-cyclodextrin, AZ-3102, Sebelipase alfa (SBC-102), Olipudase Alfa , and others.

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Niemann-Pick Type C Disease Emerging Drugs

* 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).

Niemann Pick C Disease Market Drivers

* Use of biomarkers for confirming the diagnosis of Niemann-Pick Type C Disease

Niemann Pick C Disease Market Barriers

* Undiagnosed Cases
* Less Number of Patients
* Lack of approved therapies

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The Niemann Pick C Disease pipeline report provides insights into

* The report provides detailed insights about companies that are developing therapies for the treatment of Niemann Pick C Disease 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 C Disease Treatment.
* Niemann Pick C Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Niemann Pick C Disease 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 C Disease market.

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

* Monoclonal antibody
* Small molecule
* Peptide

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Scope of the Niemann Pick C Disease Pipeline Report

* Coverage- Global
* Niemann Pick C Disease Companies- Zevra Therapeutics, Inc., Azafaros, Cyclo therapeutics, IntraBio, SOM Biotech, ENDECE and others.
* Nieman Pick C Disease Pipeline Therapies- Arimoclomol, N-Acetyl-L-Leucine, IB1001, Miglustat, Hydroxypropyl-beta-cyclodextrin, AZ-3102, Sebelipase alfa (SBC-102), Olipudase Alfa , and others.
* Niemann Pick C Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Niemann Pick C Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Content

* 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
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* AZ 3102: Azafaros
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I/II)
* Drug Name: Company Name
* Drug profiles in the detailed report.....
* 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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