Summary

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 ?-glucosidase. ?-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 -

  • ERT therapies are currently the cornerstone of PD treatment. Patients typically receive therapy from diagnosis to receive reduce glycogen accumulation. Nevertheless, ERT therapy cannot cross the blood brain barrier causing inevitable deterioration of the CNS resulting in death. Because of this, there are considerably high unmet needs within the indication. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the PD market with novel mechanisms of action?
  • The current late-stage PD pipeline encompasses one additional ERT therapy being development by the current market drug owners Sanofi Genzyme. Will the late-stage drug make a significant impact on the PD market? Will this late-stage developmental drug capture a share of the PD market? and why?



Companies mentioned: Sanofi Genzyme, Valerion Therapeutics, Amicus Therapeutics, Actus Therapeutics, Audentes Therapeutics


Scope

  • Overview of pulmonary arterial hypertension, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized PD therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2017 and forecast for ten years to 2027.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the PD therapeutics market.
  • Overview of pulmonary arterial hypertension, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
  • Annualized PD therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2017 and forecast for ten years to 2027.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the PD therapeutics market.




Reasons To Buy

The report will enable you to -

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.
  • Develop business strategies by understanding the trends shaping and driving the global PD therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global PD therapeutics market in future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Track drug sales in the global PD therapeutics market from 2017-2027.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.