Market Research Report
Pompe Disease: Opportunity Analysis and Forecasts to 2027
|Published by||GlobalData||Product code||796830|
|Published||Content info||86 Pages
Delivery time: 1-2 business days
|Pompe Disease: Opportunity Analysis and Forecasts to 2027|
|Published: November 27, 2018||Content info: 86 Pages||
Pompe disease (PD) is a rare genetic disorder resulting in the accumulation of glycogen with lysosomes in various tissues of the body. It is caused by a multitude of different phenotypical mutations to the GAA gene which is responsible for the transcription of α-glucosidase. α-glucosidase is responsible for glycogen degradation through enzymatic cleavage. PD is characterized by two types: infantile-onset Pomp disease (IOPD) and late-onset Pompe disease (LOPD). IOPD tends to be caused a severe deficiency or entire absence of GAA, resulting in symptoms being apparent from birth. Sufferers of IOPD tend to not reach their second birthday. LOPD has a large spectrum of severity, with symptoms manifesting typically in early adulthood. Sufferers have known to live a full life span if diagnosed early and given enzyme replacement therapy (ERT) to slow the progression of the disease.
It is estimated that the 2017 sales for the PD market to be approximately $684M across the 8 major markets (US, 5EU (France, Germany, Italy, Spain, and the UK), Japan, and Brazil). The US market is anticipated to grow the fastest of the three regions, recording a CAGR of 4.9%, while the 5EU and Japan will each record CAGRs of 1.2% and 0.6%, respectively.
At the end of 2027, the US will contribute around 51.9% of global sales, while the 5EU and Japan will account for 35.7% and 10.3% of global sales, respectively. The higher sales numbers for the US can be attributed to the higher price of pharmaceuticals and the greater diagnosed prevalence of PD. By the end of the forecast period in 2027, PD sales are expected to grow to $903M at a Compound Annual Growth Rate (CAGR) of 2.8% over the 10-year period. The majority of sales in the 8MM in 2027 will come from the US, which will represent 41.9% of the market.
An increase in the number of newly diagnosed cases of PD is forecasted, and consequently in the number of treatable Pompe patients, as a result of increasing awareness of PD among physicians. It is also expected that because PD is a rare, life-threatening disease with debilitating symptoms, ERT will continue to have a positive reimbursement status in the 5EU and Japan, despite the high cost, and will continue to be covered by insurance plans in the US.
The report "Pompe Disease: Opportunity Analysis and Forecasts to 2027", answers the following key questions -
Companies mentioned: Sanofi Genzyme, Valerion Therapeutics, Amicus Therapeutics, Actus Therapeutics, Audentes Therapeutics
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