Niemann Pick Disease Type C Market

About This Presentation

Title:

Niemann Pick Disease Type C Market

Description:

Niemann Pick Disease Type C is a progressive, irreversible and chronically debilitating neurovisceral lysosomal Lipid Storage Disease, which is characterized by the visceral, and neurological manifestations. – PowerPoint PPT presentation

Number of Views:88

Slides: 12

Provided by: krehani

Category: Products & Services

more less

Transcript and Presenter's Notes

Title: Niemann Pick Disease Type C Market

1
(No Transcript)
2
Niemann-Pick Disease Type C (NP-C)
It is a progressive, irreversible and chronically
debilitating neurovisceral lysosomal Lipid
Storage Disease, which is characterized by the
visceral, and neurological manifestations.
01
NP-C Classification includes Niemann Pick Type C1
and Niemann-Pick Disease Type C2. Mutation in
NP-C1 gene leads to Type C1 whereas mutation in
NP-C2 gene leads to type C2.
02
NP-C Symptoms include sudden muscle problems like
seizures, clumsiness, tremors, problems walking,
sudden falls, slurred speech and trouble in
moving eyes up and down.
03
It involves unique abnormalities of intracellular
transport of endocytosed cholesterol with
sequestration of unesterified cholesterol in
lysosomes and late endosomes.
04

DelveInsight 2019 All rights reserved

3
NP-C Epidemiology
NP-C diagnosis rate in the 7MM is assessed to be
around 50 and is expected to increase.
NP-C higher prevalence was 1011 cases in 2017 in
the US, followed by EU5 and Japan.
NP-C total prevalent population in the 7 Major
Markets (7MM) was 2115 cases in 2017.
NP-C prevalence in Western Europe was
approximately 1/120,000 to 1/150,000 living
births.
Niemann-Pick Disease Type C (NP-C) prevalence is
similar in both males and females.
7MM- USA, EU-5(Germany, Spain, Italy, France
and UK), Japan

DelveInsight 2019 All rights reserved

4
NP-C Market Outlook
Niemann-Pick Disease Type C therapeutic market in
7MM is expected to reach USD 162.30 million by
2028.
Subtype Specific NP-C Prevalence in the United
States.
5
32
Mutations in NP-C1 gene were observed in majority
of the patients whereas mutations in NP-C2 gene
were reported in nearly 5 of the patient
population In 2016.
The United States accounts for 32 of the total
sales in 2017. The United States accounts for the
largest NP-C market size, in comparison to EU5
(the United Kingdom, Germany, Italy, France, and
Spain) and Japan.

DelveInsight 2019 All rights reserved

5
NP-C Market Outlook
Growth of NP-C market could also increase due to
the introduction of diagnostic equipment. (e.g.
Biomarkers such as oxysterols help in improving
NP-C diagnosis with unclear biochemical
phenotypes and NP-C gene mutations)
Increasing healthcare expenditure, rise in RD
activities, awareness among patients about NP-C
treatment options supports NP-C market growth.
To overcome toxicity, severe side effects and
complications of NP-C current treatment methods,
many companies are developing potential
therapies for NP-C treatment.
NP-C market size shall experience a positive
growth during the forecast period (2018-2027)

DelveInsight 2019 All rights reserved

6
NP-C Emerging Drugs and Companies
Drug- VTS-270 Company- Mallinckrodt
Drug- Trappsol Cyclo Company- CTD Holdings
Drug- Arimoclomol Company- Orphazyme
Company- IntraBio

DelveInsight 2019 All rights reserved

7
Complete Information
For complete information on
Niemann-Pick Disease Type C (NP-C) Market
Insights, Epidemiology Market Forecast-2028
please click the link below
CLICK HERE
OR
Write Us At
info_at_delveinsight.com