Fda announces pilot program to treat Duching's muscular dystrophy

, Wave Life Sciences announced that it has been selected for the FDA's Innovative Complex Trial Designs (CID) pilot project to validate its Phase 2/3 clinical trial project for the treatment of Duching's muscular dystrophy (DMD) in the research drug suvodirsen (WVE-210201). It is worth mentioning that this is the first clinical trial program to be selected since the CID pilot project was launched in August 2018. Rare diseases such as DMD, often due to the scarcity of test subjects, make clinical trials difficult to carry out, so the FDA set up a CID pilot program to help modernize clinical trial design and promote drug development, with a view to benefiting patients with rare diseases as soon as possible.
progressive muscular dystrophy DMD, an X-series genetic disorder caused by a mutation in the gene encoded on the X chromosome dystrophin. The absence or defect of anti-muscular atrophy protein eventually leads to muscle fiber degradation, tissue fibrosis and premature death. DMD mainly affects men, with a prevalent rate of about 1:5000 in male infants.Suvodirsen is a stereotactic oligonucleotide that targets the antimyostrophic protein mRNA reading box, focusing on the introduction of exon skipping, designed to produce short but functional antimyostrophic proteins. According to statistics, about 13% of DMD patients carry gene mutations suitable for exon 51 jump therapy. Preclinical studies have shown that the therapy restores 52% of antimyostrophy protein function. Previously, suvodirsen was awarded FDA-issued orphan drug eligibility and a rare pediatric disease.Wave plans to recruit 40 DMD patients between the ages of 5 and 18 to conduct phase 2/3 trials. The two key criteria selected for the CID pilot project are innovative trial design features and medical needs, such as those in disease areas where there is limited or no cure. Wave's CID pilot application includes the use of historical DMD control group data to enhance the persuasiveness of the placebo group, as well as other innovative trial design elements. Given the rarity of DMD, Wave can reduce the number of trial subjects needed to provide proven clinical outcomes and potentially speed up the completion of trials.Dr Michael Panzara, Wave's chief medical officer, said: "In designing clinical trials, Wave has been looking to maximize the likelihood of determining the outcome of the trial, while combining feedback from patients and their families to reduce the burden on those who have bravely battled this serious genetic disease. The FDA's recognition of the pilot program reflects Wave's thoughtful and collaborative approach to clinical development. Wave looks forward to further discussions with the FDA in the coming months and sharing with the rare disease drug development community the knowledge learned in trial design to improve the efficiency and productivity of future clinical trials. " (Bio Valley)