At the Muscular Dystrophy Association (MDA) 2025 Conference, Roche will discuss their ongoing work on Elevidys Gene Therapy.
Late-breaking oral on Elevidys’ Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD)
Elevidys Ongoing Clinical Studies
Elevidys, EMBARK’ta (Aşama III, NCT05096221 ) tedaviden iki yıl sonra ve Çalışma 101’in (Aşama I/II, NCT03375164 , n=4), Çalışma 102’nin (Aşama II, NCT03769116 , n=26) ve ENDEAVOR Kohort 1’in (Aşama Ib, NCT04626674 , n=20) birleştirilmiş analizinde tedaviden üç yıl sonra motor fonksiyonun temel ölçümlerinde istatistiksel olarak anlamlı ve klinik olarak anlamlı iyileşmeler gösterdi, iyi eşleştirilmiş harici kontrol gruplarıyla karşılaştırıldığında. Toplu olarak, bu veriler Elevidys ile tedavinin, tedavi sırasında dört ila sekiz yaşlarındaki bireylerde hastalığın ilerlemesinin uzun vadeli stabilizasyonu veya yavaşlaması ile sonuçlandığını göstermektedir.
Elevidys showed statistically significant and clinically meaningful improvements across key measures of motor function two years after treatment in EMBARK (Phase III, NCT05096221) and three years after treatment in a pooled analysis of Study 101 ( Phase I/II, NCT03375164, n=4), Study 102 (Phase II, NCT03769116, n=26), and ENDEAVOR Cohort 1 (Phase Ib, NCT04626674, n=20), compared to well-matched external control groups. Collectively, these data demonstrate that treatment with Elevidys results in long-term stabilisation or slowing of disease progression in individuals aged four through eight years of age at the time of treatment.
Topline data from year two of the EMBARK trial were announced in January 2025. No new safety signals were observed in the EMBARK study over the two-year duration.
To further evaluate the effect of Elevidys on disease progression, muscle health and changes in muscle pathology were assessed by magnetic resonance imaging (MRI) in a subset of individuals in EMBARK part one. At week 52, results showed stabilisation or slowing of disease progression in Elevidys-treated patients compared to placebo-treated patients. At week 104, MRI changes from baseline continued to generally favour Elevidys versus placebo at week 52 across muscles and muscle groups.
Read More: Elevidys Gene Therapy: Is It Worth the Price?
Marketing Authorization for Elevidys
Elevidys is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the US, United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. Elevidys is also approved for the treatment of ambulatory individuals aged four through seven years in Brazil and Israel. Filings have also been submitted to the European Medicines Agency (EMA) and regulatory authorities in Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia. To date, more than 600 patients have been treated with Elevidys.
Learn More: Why Isn’t Roche Applying Marketing Authorization in Every Country for Elevidys Gene Therapy?
