Niemann Pick C Disease- Pipeline Insight, 2025

This “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 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.

Niemann Pick C Disease: Understanding

Niemann Pick C Disease: Overview

Niemann-Pick Disease Type C (NP-C) is a progressive, irreversible, pan-ethnic and chronically debilitating neurovisceral Lysosomal Lipid Storage Disease which is characterized by the visceral, neurological and psychiatric manifestations. It involves the unique abnormalities of intracellular transport of endocytosed cholesterol with sequestration of unesterified cholesterol in lysosomes and late endosomes. It occurs due to the alterations in trafficking of endocytosed cholesterol. It ultimately results in the accumulation of the cholesterol and glycosphingolipids in various neurovisceral tissues. At the cellular level, Niemann Pick Type C can be caused by two different genetic mutations. Type C1 is caused by a mutation in NPC1 gene and type C2 is caused by a mutation in the NPC2 gene. NPC1 is found in the majority of cases as compared to NPC2. There is considerable variation when type C symptoms first appear (enlarged liver, enlarged spleen, or jaundice) and the progression of the disease. These symptoms usually appear as early as a few months of age or as late as childhood. Symptoms include sudden muscle problems such as seizures, clumsiness, tremors, problems walking, sudden falls, slurred speech and trouble moving eyes up and down. In the majority of the cases, the neurological symptoms begin appearing in the ages of 4 to 10. As the disease progresses, the children start developing learning disabilities, psychological problems, or even dementia, and often lose the ability to speak.

Niemann-Pick Disease Type C is associated with the mutations of NPC1 and NPC2 genes. No deficiency of sphingomyelinase is observed among the patients with Niemann-Pick Disease Type C. Mutations in NPC1 and NPC2 genes lead to severe impairment of intracellular lipid transport due to the sequestering of endocytosed LDL-derived cholesterol leading to the impaired cellular trafficking. Transport of perinuclear lysosomes to the cell membrane is hindered which also results in the accumulation of unesterified cholesterols, phospholipids, and glycosphingolipids in liver and spleen. Medical history and general physical examination, biochemical testing, genetic testing, bone marrow biopsy, biomarkers are used for the diagnosis of Niemann-Pick Disease Type C. Cell biology is done for studying intracellular trafficking of LDL-derived cholesterol. Filipin test, Cholesterol esterification test and for wider screening for NPC, there is a need for convenient blood-based, cost-effective, and if possible, specific biomarkers. Bone marrow biopsy and genetic testing is done for the diagnosis of the disease.

Niemann-Pick Disease Type C can be treated by using disease-specific therapies and symptomatic therapies are recommended for treating neurological, visceral and psychiatric manifestations associated with Niemann-Pick Disease Type C. The common therapies used for treatment purpose are Cholesterol-lowering drugs, Sphingolipid-lowering drugs that includes, Sterol-binding drug therapy, INPC replacement therapy, and Alternative therapies. The primary purpose of the therapies is to reduce the progression of the disease, management of the disease-specific manifestations and managing the signs and symptoms.

'Niemann Pick C Disease- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann Pick C Disease pipeline landscape is provided which includes the disease overview and Niemann Pick C Disease treatment guidelines. The assessment part of the report embraces, in depth Niemann Pick C Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Niemann Pick C Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Niemann Pick C Disease R&D. The therapies under development are focused on novel approaches to treat/improve Niemann Pick C Disease.

Niemann Pick C Disease Emerging Drugs Chapters

This segment of the Niemann Pick C Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Niemann Pick 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, 2024. 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: Therapeutic Assessment

This segment of the report provides insights about the different Niemann Pick C Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Niemann Pick C Disease

There are approx. 10+ key companies which are developing the therapies for Niemann Pick C Disease. The companies which have their Niemann Pick C Disease drug candidates in the most advanced stage, i.e. Registration include, Zevra Therapeutics, Inc

Phases

The report covers around 15+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

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

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Niemann Pick C Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann Pick C Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Niemann Pick C Disease drugs.

Niemann Pick C Disease Report Insights

• Niemann Pick C Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Niemann Pick C Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Niemann Pick C Disease drugs?
• How many Niemann Pick C Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Niemann Pick C Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Niemann Pick C Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Niemann Pick C Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Zevra Therapeutics, Inc
• Azafaros
• Cyclo therapeutics
• IntraBio
• SOM Biotech
• ENDECE

Key Products

• Arimoclomol
• AZ 3102
• Trappsol Cyclo
• IB1001
• SOM-0208
• NDC 1308

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