The next wave of Duchenne trials
Five-plus BLA submissions are expected in 2026. For sponsors, the bottleneck is rarely the science — it is finding sites that can enrol enough patients, fast enough, without compromising data quality.
Duchenne muscular dystrophy (DMD) remains one of the most closely watched areas in rare disease drug development. As the current wave of gene therapies, exon-skipping candidates and disease-modifying treatments moves through late-stage trials, several sponsors are expected to file Biologics License Applications in 2026. Each of those filings depends on enrolment numbers that, in many established trial markets, are becoming harder to hit.
Where the patients are
Armenia, Georgia, Ukraine and Uzbekistan represent a combined population of roughly 75 million people. Our modelling puts the number of DMD patients across these four countries at over 1,400 — the large majority of them treatment-naïve. For a disease this rare, that is a meaningful, largely untapped pool of eligible participants.
Why the bottleneck is operational, not scientific
Running a rare disease trial in a new region raises real questions: site readiness, regulatory familiarity with the protocol, genetic testing infrastructure, and the ability to keep families engaged over what can be a multi-year study. These are solvable problems with the right operational partner on the ground — which is the gap we built our CEE and Central Asian network to close.
What this means for sponsors planning 2026 enrolment
For sponsors weighing where to add sites ahead of a 2026 filing, the calculus is straightforward: a treatment-naïve population removes the competing-trial pressure that slows enrolment in more saturated markets, provided the operational groundwork — site selection, ethics submissions, patient identification — is done early.
Want the full modelling behind these numbers, broken down by country? Get in touch and we'll walk you through it.