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The global treatment market for Duchenne Muscular Dystrophy (DMD) will expand in value at a staggering Compound Annual Growth Rate (CAGR) of 160.5 percent, from approximately $8.2 million in 2014 to $990 million by 2019, says research and consulting firm GlobalData.
The company's latest report states that this growth will occur across six major markets: the US, France, Germany, Italy, Spain and the UK.
Combined, the five European countries will contribute 55.2 percent to the total market value by 2019, while the US will account for 44.8 percent.
According to Dr Nikhilesh Sanyal, GlobalData's analyst covering immunology, the primary driver behind this impressive expansion will be the market entry of PTC Therapeutics' Translarna, Sarepta Therapeutics' eteplirsen, and BioMarin/Prosensa's drisapersen.
Dr Sanyal says: "These mutation-specific drugs are expected to have a high price point due to their novelty, efficacy and orphan drug status, and will contribute 85.6 percent to the DMD treatment arena by 2019. While Translarna is expected to be the top-selling DMD drug by 2019, exon-51-skipping therapies drisapersen and eteplirsen will also be a hotspot for growth, thanks to their rapid clinical development and significant demand.
"Despite rapid developments in DMD therapeutics over the last five years, there remains a large unmet need among patients who are not amenable to these mutation-specific drugs, and who would benefit from effective disease-modifying therapies."
However, the analyst adds that drug developers will need to overcome a number of challenges to take advantage of this major opportunity.
He explains: "There is currently a lack of standardization for measuring clinical efficacy across all stages of DMD. While most studies use the change in the six-minute walk test as the primary clinical endpoint, this applies only to ambulant patients and therefore excludes non-ambulant individuals, as well as children under the age of five.
"Furthermore, the high cost of novel disease-modifying drugs means that their reimbursement by local health authorities and insurance companies may be prevented following their entry into the arena," the analyst concludes.
