- The author 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, and a positive CHMP opinion.
- There were 13 licensing and asset acquisition activities involving Duchenne muscular dystrophy drugs during 2013-17, six of which occurred in 2017. The $562m license and collaboration agreement between Sarepta Therapeutics and Summit Therapeutics for European rights to Summit’s utrophin modulator pipeline for the treatment of Duchenne muscular dystrophy was the largest deal during the period.
- 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 57.5% of trials in Phase II, and only 35% and 7.5% of trials in Phase III and I, respectively.
- The US has a substantial lead in the number of Duchenne muscular dystrophy clinical trials globally. Italy 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.
Vitamin D supplement
Approvals by country
KEY UPCOMING EVENTS
KEY REGULATORY EVENTS
Exondys Revisited? Translarna Brings Efficacy Woes Into US Panel Review
Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
Sarepta's Exondys 51 (Eteplirsen) Approved After Contentious Internal FDA Debate
Marathon Looking To Go The Distance In DMD
Scots Hammer PTC's Translarna As EU Reviews Conditional Approval
FDA Refuses To File Translarna NDA; PTC Plummets
LICENSING AND ASSET ACQUISITION DEALS
Sarepta Options Duke's CRISPR/Cas9 IP For DMD
Lucky No. 35? Sarepta And BioMarin Settle Global Patent Disputes
Bristol Licenses Phase II DMD Candidate To Roche
Sarepta Snaps Up Gene Therapy Approaches to DMD
Sarepta’s $852m Summit Collaboration Could Enable DMD Drug Combinations
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Summit DMD Data Intrigue But Longer-Term Results Awaited
Capricor's DMD Cell Therapy Has HOPE, But Market Is Unsure
List of Figures
Figure 1: Trends in prevalent cases of Duchenne muscular dystrophy, 2016-25
Figure 2: Overview of pipeline drugs for Duchenne muscular dystrophy in the US
Figure 3: Pipeline drugs for Duchenne muscular dystrophy, by company
Figure 4: Pipeline drugs for Duchenne muscular dystrophy, by drug type
Figure 5: Pipeline drugs for Duchenne muscular dystrophy, by classifications
Figure 6: Key upcoming events in Duchenne muscular dystrophy
Figure 7: Licensing and asset acquisition deals in Duchenne muscular dystrophy, 2013-17
Figure 8: Parent patents in Duchenne muscular dystrophy
Figure 9: Clinical trials in Duchenne muscular dystrophy
Figure 10: Top 10 drugs for clinical trials in Duchenne muscular dystrophy
Figure 11: Top 10 companies for clinical trials in Duchenne muscular dystrophy
Figure 12: Trial locations in Duchenne muscular dystrophy
Figure 13: Duchenne muscular dystrophy trials status
Figure 14: Duchenne muscular dystrophy trials sponsors, by phase
List of Tables
Table 1: Prevalent cases of Duchenne muscular dystrophy, 2016-25
Table 2: Marketed drugs for Duchenne muscular dystrophy
Table 3: Approvals by country for Duchenne muscular dystrophy
Table 4: Pipeline drugs for Duchenne muscular dystrophy in the US
Table 5: Historical global sales, by drug ($m), 2012-16
Table 6: Forecasted global sales, by drug ($m), 2017-22