Niemann Pick Disease Drug Type C Treatment Market

Niemann-Pick Type C (NPC) is a rare inherited disease. This type of genetic mutation causes cholesterol and other fats to build up in the liver, spleen, or lungs. It has an impact on the brain as well. The onset of symptoms for NPC may begin at different ages. It has been grouped as: infantile (after birth to less than 6 years), juvenile (6 to 15 years), and adults (15 years to greater). In infants, NPC leads to disorders such as alpha-1-antitrypsin deficiency, tyrosinemia, Gaucher disease, idiopathic neonatal cholestasis, and various congenital infections.

Market Dynamics

Increasing government initiatives for rare diseases is expected to foster global niemann-pick disease type C treatment market growth over the forecast Period. For instance, in November 2021, the Italian senate unanimously approved the consolidated act on rare diseases. The aim of the act is to make the treatment of rare diseases uniform throughout the Italian territory and to promote progress in research. Moreover, the act also provides for the establishment of solidarity fund for people with rare diseases, with an initial budget of US$ 1.01 million per year starting from 2022.

Rising numbers of collaborations and agreements by market players are expected to drive growth of the global Niemann-pick disease drug type C treatment market over the forecast period. For instance, in May 2022, Kempharm, Inc., a specialty pharmaceutical company, announced an agreement with ORPHAZYME A/S, a global biopharmaceutical company, to acquire arimoclomol, an orally-delivered, first-in-class heat shock protein (HSP) amplifier being developed as a treatment for Niemann-Pick disease type C (NPC)

Increasing research and development activities by market players are expected to drive growth of market over the forecast period. For instance, in June 2021, Cyclo Therapeutics, Inc., a clinical-stage biotechnology company, announced the commencement of its pivotal Phase 3 study (TransportNPC) for the evaluation of Trappsol Cyclo, a proprietary formulation of hydroxypropyl beta cyclodextrin, delivered intravenously, for treating Niemann-Pick Disease Type C1 (NPC1). The TransportNPC study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg of Trappsol Cyclo administered intravenously and standard of care (SOC) compared to placebo administered intravenously and SOC in patients with NPC1.

Key features of the study:

• This report provides an in-depth analysis of the global Niemann-pick disease drug type C treatment market , market size (US$ Million), and compound annual growth rate (CAGR%) for the forecast period (2022-2030), considering 2021 as the base year
• It elucidates potential revenue opportunities across different segments and explains an attractive investment proposition matrix for this market.
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategies adopted by the leading players
• It profiles leading players in the global Niemann-pick disease drug type C treatment market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies, and future plans.
• Key companies covered as a part of this study include Livewise Johnson & Johnson, Intrabio, Cyclo Therapeutics, Inc., Kempharm, Inc., Azafaros B.V., StrideBio. Sarepta Therapeutics, Inc.
• Insights from this report would allow marketers and the management authorities of the companies to make an informed decision regarding their future product launch, technology up-gradation, market expansion, and marketing tactics.
• The global Niemann-pick disease drug type C treatment market report caters to various stakeholders in this industry including investors, suppliers, manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision making through various strategy matrices used in analyzing the global Niemann-pick disease drug type C treatment market

Detailed Segmentation:

• Global Niemann-Pick Disease Drug Type C Treatment Market, By Drug Type:
  • Phase III Drug
    • Trappsol Cyclo
    • IB1001
  • Marketed Drug
    • Miglustat (Zavesca)
• Global Niemann-Pick Disease Drug Type C Treatment Market, By Indication:
  • Niemann-Pick Disease Drug Type C1
  • Niemann-Pick Disease Drug Type C2
• Global Niemann-Pick Disease Drug Type C Treatment Market, By Distribution Channel:
  • Hospital Pharmacies
  • Online Pharmacies
  • Retail Pharmacies
• Global Niemann-Pick Disease Drug Type C Treatment Market, By Region:
  • North America
    • By Country:
      • U.S.
      • Canada
  • Latin America
    • By Country:
      • Brazil
      • Mexico
      • Argentina
      • Rest of Latin America
  • Europe
    • By Country:
      • U.K.
      • Germany
      • Italy
      • Spain
      • France
      • Russia
      • Rest of Europe
  • Asia Pacific
    • By Country
      • China
      • India
      • Japan
      • Australia
      • South Korea
      • ASEAN
      • Rest of Asia Pacific
  • Middle East
    • By Country:
      • GCC
      • Israel
      • Rest of Middle East
  • Africa
    • By Country/Region:
      • South Africa
      • Central Africa
      • North Africa
• Company Profiles
  • Johnson & Johnson *
    • Company Overview
    • Product Portfolio
    • Financial Performance
    • Recent Developments/Updates
    • Strategies
  • Intrabio
  • Cyclo Therapeutics, Inc.
  • Kempharm, Inc
  • Azafaros B.V.
  • StrideBio
  • Sarepta Therapeutics, Inc.

 “*” marked represents similar segmentation in other categories in the respective section.