Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin. It is an X-linked recessive disorder mainly affecting males, that causes muscles to be fragile and easily damaged. DMD is progressive and most affected individuals require a wheelchair by adolescence.

GlobalData’s latest report “Duchenne Muscular Dystrophy: Opportunity Assessment and Forecast to 2030” covers the US and Japanese markets and provides a forecast from 2020-2030.

Chris Pilis, Immunology Analyst at GlobalData, comments: “The DMD market in the US and Japan was valued at $619.0M in 2020 of which the US comprised 98%. Current treatments in the US and Japanese markets consist of exon-skipping therapies and steroids. In the US there are currently four exon-skipping therapies, three of which are manufactured by Sarepta Therapeutics, and two steroid therapies, namely PTC Therapeutics’ Emflaza (deflazacort) and highly genericized prednisone, whereas only two treatments are available in Japan, Nippon Shinyaku’s Viltepso (exon-skipping therapy) and highly genericized prednisone.

Greatest unmet needs in the DMD space pertain to disease progression rate, access to treatment for specific patient segments, and cost of therapies. Current treatments can neither stop disease progression nor improve patients’ health status and thus decline of vital functions is inevitable. Loss of ambulation leads physicians to discontinue exon-skipping therapy for patients with amenable mutations, leaving steroids as the only treatment option for this patient subset. Aside from prednisone, current DMD therapies are very expensive and pipeline candidates are not expected to be priced lower than currently marketed products.

Pilis adds: “The DMD pipeline consists of 23 drugs across all stages of development in the US and Japan with eight products in late-stage development, of which two are gene therapies, one is a cell therapy, one is a monoclonal antibody, and four are small molecules.”

Pilis concludes: “The market is projected to grow at a Compound Annual Growth Rate of 12.0%, reaching $1.9B by 2030. The sales of high-priced exon-skipping therapies and Emflaza (deflazacort) represent the vast majority of the DMD market, with exon-skipping therapies holding over three-quarters of the market.

Very expensive pipeline therapies are anticipated to dominate the market by 2030, reducing the market share of current treatments to about one-third.

GlobalData expects gene therapies to shift the competitive landscape in the DMD market, by potentially providing treatment with a single administration and by covering patients with more mutations compared to exon-skipping therapies. However, gene therapies will be more expensive than current treatments, thus it is likely that only patients that meet the enrollment criteria of clinical trials will be eligible for this type of treatment, including patients between four and seven years of age who are ambulatory.”