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:
Detailed Segmentation:
“*” marked represents similar segmentation in other categories in the respective section.
Table of Contents
*Key pointers provided are also applicable to other companies covered in the scope of the report.
*Browse 35 market data tables and 29 figures on “Global Niemann-Pick Disease Drug Type C Treatment Market” - Forecast to 2030.
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