REGENXBIO is set to apply for US accelerated approval of its Duchenne muscular dystrophy (DMD) gene therapy after it showed benefit in a Phase III trial.
The Phase III portion of the AFFINITY DUCHENNEtrial, (NCT05693142), investigating RGX-202, met its primary endpoint with high statistical significance, with 93% of participants reaching at least 10% microdystrophin expression after 12 weeks.
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Additionally, RGX-202 demonstrated statistically significant correlation between microdystrophin expression and interim functional improvement. Microdystrophin expression averaged 71.1% across all patients, and 41.6% in older boys, aged >8 years. Additionally, 80% of participants achieved >40% microdystrophin expression.
After one year, patients treated with RGX-202 demonstrated functional improvement and evidence of positively impacting disease trajectory as measured by North Star Ambulatory Assessment (NSAA) and timed function tests (Time to Stand, 10 Meter Walk-run, Time to Climb).
Pat Furlong, founding President of Parent Project for Muscular Dystrophy, said: “For decades, our community has pushed for therapies that can change the trajectory of this disease, and today’s news gives us renewed optimism. Our families cannot wait; regulatory flexibility for innovative medicines to treat rare disease remains an urgent priority. We applaud the dedication of the patients and families who participated in this research and look forward to continued progress toward delivering stronger futures for people with Duchenne.”
The data from RGX-202 shows higher benefit than Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl), which saw microdystrophin expression levels of 34.29%, compared to 71.1% with REGENXBIO’s therapy. However, a head-to-head study is yet to be conducted.
Overall, RGX-202 was well tolerated and demonstrated a favourable safety profile. There were, however, two serious adverse events were reported; both were easily managed and resolved within weeks without sequelae.
One case of subacute myocarditis was reported in an eight-year-old patient, while one 10-year-old patient suffered a case of asymptomatic liver injury.
Based on the study data, REGENXBIO will discuss the data with the US Food and Drug Administration (FDA) at a future meeting with plans to pursue accelerated approval for RGX-202 and is preparing for a potential commercial launch in 2027.
The company is also finalising the trial design for an ex-US study to support global regulatory submissions.
On the same day as releasing its trial data, REGENXBIO also posted underwhelming Q1 earnings, causing the company’s stock to drop by 37.8%, from a 13 May close of $10.04 to a 14 May close of $6.24.
According to a GlobalData report, the DMD market across the seven major markets (7MM: US, France, Germany, Italy, Spain, the UK, and Japan) is set to grow from $2.3bn in 2023 to $5.2bn.
Asiyah Nawab, Healthcare Analyst at GlobalData, comments: “The DMD treatment landscape is evolving with the emergence of novel therapies such as exon-skipping and gene therapies. However, gene therapies in particular, compared to exon-skipping, will have less of an impact due to the small patient share eligible for treatment, in addition to the high cost of these medicines limiting patients’ access. By 2033, GlobalData forecasts gene therapies to contribute $821m to the DMD market, a lower figure relative to exon-skipping therapies.”
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Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
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