The trial aimed to test the safety and effectiveness of Resolaris in people with FSHD who were diagnosed before 10 years of age. Eight people aged between 16-20 took part in the 12-week study.
The results showed that Resolaris was safe and well-tolerated, including at the highest dose. 63% of participants showed an improvement in muscle strength and 67% reported an improvement in their quality of life.
Although this trial had small numbers of participants, its findings are promising. They also confirm what was observed in a separate phase 1/2 trial, where 50% of adults with FSHD (not early-onset) had improvements in muscle function.
Dr Sanjay Shukla, aTyr Pharma’s Chief Medical Officer, said:
These results are important as they reinforce previous clinical results (in adult FSHD and adult LGMD2B) with Resolaris in a younger patient population, with a potentially more aggressive progression of disease.
Resolaris has Orphan Drug Designation for the treatment of both FSHD and limb girdle muscular dystrophy (LGMD). This special status gives companies certain incentives to help develop and market their rare disease product.
Read the latest FSHD research news
Find out about FSHD research that we’re funding