Niemann-Pick disease type C Market

Niemann-Pick disease type C Overview

Niemann–Pick disease type C is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside the cells. This inability leads to the abnormal accumulation of these substances within various tissues of the body and brain.

Niemann–Pick disease type C varies according to the different age of onset and Niemann–Pick disease type C symptoms can vary from one person to another person. This genetic disorder characterized by visceral, neurological, and psychiatric manifestations. At the cellular level, Niemann–Pick disease type C can be caused by a mutation in the NPC1 gene or NPC2 gene and is inherited in an autosomal recessive manner. Niemann–Pick disease type C has an extremely heterogeneous clinical presentation, and there is considerable variation when type C symptoms first appear (enlarged liver, enlarged spleen, or jaundice) and the progression of the disease. Niemann–Pick disease type C diagnosis is based on the characteristic symptoms like psychiatric issues, neurologic findings, and many others. Sometimes this disorder is misdiagnosed for other associated neurological disorder. Niemann–Pick disease type C affects males and females equally.

Niemann-Pick disease type C Epidemiology

Niemann–Pick disease type C total prevalent population in the 7MM was observed to be 2116 in 2017. Niemann–Pick disease type C epidemiology model estimates that lysosomal storage disease subtype is the most prevalent in the 7MM countries when compared to Niemann–Pick disease type C2 subtype. Niemann-Pick disease type C highest prevalence in the United States with 1011 cases in 2017. Moreover, among the European 5 countries, Germany had the highest Niemann–Pick disease type C diagnosed prevalent population with 251 cases in 2017, and then comes France and the United Kingdom. Whereas, Spain had the lowest Niemann–Pick disease type C diagnosed prevalent population of 151 in 2017. Moreover, Japan witnessed 102 diagnosed Niemann–Pick disease type C prevalent cases in 2017. The total diagnosed Niemann–Pick disease type C prevalent population in the 7MM was assessed to be 1,058 in 2017. Niemann–Pick disease type C Epidemiology model is based on disease phenotypes, indicating the total Niemann–Pick disease type C cases in the 7MM. In the United States, juvenile cases makes up the majority of the patient population, on the other hand, in the United Kingdom, most commonly occurring was observed to be Early infantile.

Niemann-Pick disease type C Market

Taking into the consideration of the therapeutic Niemann-Pick disease type C market in the 7MM, it was analysed to be USD 28.676 million in 2017 and is anticipated to rise during the study period (2017-2028). Niemann-Pick disease type C market share of prescription therapies was found to be USD 9.254 million in 2017, in the United States. This was entirely dependent on supportive treatment regimens. Of the supportive treatment regimens, Zavesca, which has already been approved in the EU-5 countries and Japan, contributed USD 9.214 million in 2017 in the United States. In contrast to the US therapeutic Niemann-Pick disease type C market, the current therapeutic Niemann–Pick disease type C market size in the EU-5 countries, and Japan got control with the use of only approved drug Zavesca/Brazaves [Miglustat; by Actelion] and the supportive therapies.

Niemann-Pick disease type C Emerging Therapies

There are many emerging therapies that are developed by many biopharmaceutical companies like VTS-270 (by Mallinckrodt), Trappsol Cyclo (by CTD Holdings), and Arimoclomol (by Orphazyme) that are expected to enter Niemann-Pick disease type C treatment market, with their respective products, during the forecast period [2019–2028]. The only approved therapy so far [in EU-5 countries and Japan], i.e., Zavesca [Miglustat], a substrate reduction therapy developed by Actelion Pharmaceuticals, has denied the approval for Niemann-Pick disease type treatment by the US Food and Drug Administration (FDA). Arimoclomol (Orphazyme), Trappsol Cyclo (CTD Holdings), and others are have shown positive effects to cure the lysosomal disease and in coming years with the launch of this drug Niemann-Pick disease type market will see potential growth. Zavesca is the only approved drug for the Niemann-Pick disease type in Europe and Japan. More advanced research and development are required to develop a potent drug with high safety and efficacy.

There are other many supportive therapies available for the management of Niemann-Pick disease type C symptoms. These therapies mainly involve Antiepileptics (Seizures); Tricyclic Antidepressants & CNS Stimulants (Cataplexy); Anticholinergics, Trihexyphenidyl & GABA Derivatives (Dystonia & Tremor); atypical antipsychotics (Psychosis). They account for a minimal towards the Niemann-Pick disease type C market revenue.

However, progress in the novel drug development by many pharmaceutical companies lead to the dynamics of Niemann-Pick disease type C market to change in the coming years due to the expected launch of emerging therapies during the forecasted period of 2019-2028.