Reports world wide Duchenne Muscular Dystrophy (DMD)

Market Spotlight: Duchenne Muscular Dystrophy (DMD)
This Market Spotlight report covers the Duchenne muscular dystrophy market, comprising
key pipeline and marketed drugs, clinical trials, upcoming and regulatory events, patent
information, a 10-year disease prevalence forecast, and licensing and acquisition deals, as
well as presenting drug-specific revenue forecasts.
Key Takeaways
Datamonitor Healthcare estimates that in 2016, there were 179,200 prevalent cases of
Duchenne muscular dystrophy in males worldwide, and forecasts that number to increase to
196,200 prevalent cases by 2025.
Marketed drugs include US FDA-approved drugs such as Emflaza (deflazacort), a
glucocorticoid with anti-inflammatory and immunosuppressant properties, and Exondys 51
(eteplirsen), a novel phosphorodiamidate morpholino oligomer designed to induce the
skipping of exon 51 in the dystrophin gene. The marketed drugs also include an EU-
approved drug, Translarna (ataluren), which acts by targeting premature nonsense
mutations.
Therapies in mid- and late-stage development for Duchenne muscular dystrophy focus on a
variety of targets. The majority of pipeline drugs in mid- and late-stage development for
Duchenne muscular dystrophy are administered orally, with the remainder including
intravenous, intramuscular, intra-arterial, and subcutaneous formulations.
High-impact upcoming events for drugs in the Duchenne muscular dystrophy space include
topline Phase II and Phase III trial results, an estimated PDUFA, and a positive CHMP
opinion.
There were 14 licensing and asset acquisition activities involving Duchenne muscular
dystrophy drugs during 2012–17, six of which occurred in 2017. The $730m R&D
collaboration agreement between Astellas and Mitokyne (now Mitobridge) to discover and
develop compounds that target mitochondrial function was the largest deal during 2012–17.
The clinical trials distribution across Phase I–III indicates that the majority of trials for
Duchenne muscular dystrophy have been in Phase II development, with 51.1% of trials in
Phase II, and only 39.4% and 9.1% trials in Phase III and I, respectively.
The US has a substantial lead in the number of Duchenne muscular dystrophy clinical trials
globally. Belgium leads the major EU markets, while Israel has the top spot in Asia.
Clinical trial activity in the Duchenne muscular dystrophy space is dominated by ongoing
trials. PTC Therapeutics has the highest number of ongoing and completed clinical trials for
Duchenne muscular dystrophy.
PTC Therapeutics leads industry sponsors with the highest number of clinical trials for
Duchenne muscular dystrophy, followed by GlaxoSmithKline