Press release
Niemann Pick C Disease Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies
Explore the comprehensive insights by DelveInsight and stay ahead in understanding the Niemann Pick C Disease Treatment Landscape. Click here to read more @ Niemann Pick C Disease Pipeline Outlook [https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
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.
Stay informed about the cutting-edge advancements in Niemann Pick C Disease Treatments. Download for updates and be a part of the revolution in Neurology care @ Niemann Pick C Disease Clinical Trials Assessment [https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
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
Get a detailed analysis of the latest innovations in the Niemann Pick C Disease pipeline. Explore DelveInsight's expert-driven report today! @ Niemann Pick C Disease Unmet Needs [https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
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
Discover the latest advancements in Niemann Pick C Disease Treatment by visiting our website. Stay informed about how we're transforming the future of neurology @ Niemann Pick C Disease Market Drivers and Barriers, and Future Perspectives [https://www.delveinsight.com/sample-request/niemann-pick-type-c-disease-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=ypr]
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
For a detailed overview of our latest research findings and future plans, read the full details of Niemann Pick C Disease Pipeline on our website @ Niemann Pick C Disease Emerging Drugs and 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 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
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=niemann-pick-c-disease-clinical-trials-and-studies-2025-ema-pdma-fda-approvals-mechanism-of-action-roa-nda-ind-and-companies]
Phone: 09650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/report-store/niemann-pick-disease-type-c-npc-market-insights-epidemiology-and-market-forecast
Legal Disclaimer: Information contained on this page is provided by an independent third-party content provider. ABNewswire makes no warranties or responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you are affiliated with this article or have any complaints or copyright issues related to this article and would like it to be removed, please contact retract@swscontact.com
This release was published on openPR.
Permanent link to this press release:
Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.
You can edit or delete your press release Niemann Pick C Disease Clinical Trials and Studies 2025: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies here
News-ID: 3991335 • Views: …
More Releases from ABNewswire
Understanding Magnetic Switches: Types, Working Principles, and Applications
A magnetic switch is a versatile electrical device that operates by responding to magnetic fields. From Hall Effect to reed switches, explore their functions, types, and diverse applications.
What is a Magnetic Switch?
A magnetic switch is an electrical device that operates by responding to the presence of a magnetic field. It functions by closing or opening its contacts based on the strength of the magnetic field. Specifically, the switch remains closed…
Innovating embedded downlight technology: Leading the global trend of intelligen …
The strong rise of China's manufacturing industry has profoundly reshaped the technological landscape and development trajectory of embedded downlights. With its unparalleled industrial chain advantages, continuous technological innovation, and significant cost-effectiveness, Chinese enterprises have risen from participants in the global lighting industry to undisputed leaders. This transformation goes far beyond the huge production scale; it lies in the thorough innovation of the core technical content and application boundaries of embedded…
North China Electric Power University Foreign Aid Training Program Visits Yiyuan …
On July 23, 2025, the Electrical Industry Training Program of North China Electric Power University's Foreign Aid Project visited Yiyuan Technology for an exchange visit. The purpose was to gain in-depth insights into the company's innovations in renewable energy technology promotion and application [https://www.yiyen.com/about/], and to build bridges connecting university-enterprise cooperation with foreign aid initiatives.
Image: https://ecdn6.globalso.com/upload/p/1108/image_other/2025-07/scene-1.jpg
Image: https://ecdn6.globalso.com/upload/p/1108/image_other/2025-07/scene-3.jpg
Upon arrival, the delegation received a warm welcome from company leadership and the reception…
Pure Sine Wave Low Frequency Inverter Charger
Pure sine wave low frequency inverter [https://www.yiypower.com/ap-mini-600w-1500w-120v-low-frequency-pure-sine-wave-inverter-charger-product/] is widely used and suitable for a variety of scenarios. In terms of home backup power, it can provide reliable power during power outages, ensuring that essential appliances such as refrigerators, air conditioners and medical equipment continue to operate . For solar power generation systems, Inverter charger [https://www.yiypower.com/ap-mini-600w-1500w-120v-low-frequency-pure-sine-wave-inverter-charger-product/] can converter DC Power from Battery, which charged by solar panels, into clean alternating current…
More Releases for Pick
SONIC GLOW PICK REVIEWS 2024;IS SONIC GLOW PICK LEGIT OR JUST A HYPE?
The Sonic Glow Pick Has rapidly gained popularity among consumers looking for an effective solution to at-home oral care. In a world where dental hygiene is essential, but costly dentist visits can be a barrier, this innovative device provides a convenient alternative for removing plaque and tartar from your teeth without the discomfort of traditional methods. By combining advanced ultrasonic technology with user-friendly design, the Sonic Glow Pick offers professional-level…
Digital Pick System Market is Booming Across the Globe: AIOI-SYSTEMS, Wynright, …
The Latest Study Published by HTF MI Research on the "Digital Pick System Market'' evaluates market size, trend and forecast to 2029. The Digital Pick System market study includes significant research data and evidences to be a practical resource document for managers and analysts is, industry experts and other key people to have an easily accessible and self-analysed study to help understand market trends, growth drivers, opportunities and upcoming challenges…
Sonic Glow Pick Review 2023;(Shocking info!) Unique Tooth and Gum Pick Reviewed
Sonic Glow Pick is a small, slender, pointed object primarily used for cleaning and removing food particles from between teeth. It is typically made from wood, plastic, or bamboo.
Sonic Glow Picks are designed with convenience and hygiene in mind.
Sonic Glow Pick can be more efficient than traditional toothpicks for removing plaque and debris from hard-to-reach areas.
They often offer additional features such as timers, pressure settings, and interchangeable heads…
Pick-to-Light System Market 2023-2030 Revenues Will Increase Dramatically With H …
WMR has recently published a new research report titled "Pick-to-Light System Market - Global Outlook and Forecast till 2030." This report offers a comprehensive analysis of the market, drawing insights from historical milestones and current trends. It presents a detailed examination of production volumes for each type from 2018 to 2030, along with regional production volume data for the same period.
The research report provides a thorough assessment of the various…
Ortho Rinse Pick Reviews - Does Ortho Rinse Pick Really Work? Read OrthoRinse Pi …
Ortho Rinse Pick is a revolutionary oral hygiene tool that aims to enhance the oral care routine, particularly for individuals with orthodontic appliances such as braces, aligners, or retainers. This innovative device is designed to effectively clean hard-to-reach areas, ensuring optimal oral health during orthodontic treatment.
Orthodontic appliances often pose challenges when it comes to maintaining proper oral hygiene. The brackets, wires, and other components create intricate spaces where food particles,…
OrthoRinse Pick Review: (Fake Or Legit!) Is OrthoRinse Pick Worth Buying?
OrthoRinse Pick has an average rating of 4.9 out of 5 stars across all reviews from customers, and it has already received attention from more than 8,750 users. Read below to get all the necessary information you need about Ortho Rinse Pick Review.
An estimated 74 million Americans have no dental insurance coverage and I'm one of them. Regular checkups only happen twice a year, but you never know what…
