• 11 Jun 2021
• Admin
• News Article

Us Fda Accepts Dynacures Ind For Dyn101 To Treat Myotubular And Centronuclear Myopathies

Dynacure, A Clinical-Stage Company, Announced That The Us Food And Drug Administration (Fda) Has Accepted Its Investigational New Drug (Ind) Application For Dyn101, An Investigational Antisense Product Candidate Designed To Reduce The Expression Of Dynamin 2 Protein (Dnm2), For The Treatment Of Myotubular And Centronuclear Myopathies (Cnm). Dyn101 Is Currently Being Evaluated In A Phase 1/2 Clinical Trial, Unite-Cnm (Dyn101-C101), At Multiple Clinical Sites In Europe. Dyn101 Has Been Granted Orphan Drug Designation By The Us Fda And European Medicines Agency (Ema) And Rare Pediatric Disease Designation By The Us Fda. There Is Currently No Fda- Or Ema-Approved Therapeutic Treatment For Cnm."The Acceptance Of Our Ind For Dyn101 Builds Upon The Clinical Development Path We Are Following In The Eu And Will Implement In The Us With Our Ongoing Unite-Cnm Trial," Said Leen Thielemans, Chief Development Officer Of Dynacure. "There Remains Significant Unmet Medical Need For The Treatment Of Cnm And We Look Forward To Expanding Our Phase 1/2 Clinical Trial Of Dyn101 Into The United States.""Us Fda Acceptance Of The Ind For Dyn101 Significantly Advances Our Clinical Development Of Dyn101 By Expanding Our Trial Into The United States And Underscores Our Commitment To Deliver A Novel Medicine For Cnm Patients Around The World," Added Stephane Van Rooijen, Chief Executive Officer Of Dynacure.Myotubular And Centronuclear Myopathies (Cnm) Are Serious, Rare, Life-Threatening Disorders That Affect Skeletal Muscles From Birth. Cnm Derives Its Name Based On The Central Location Of The Muscle Fiber Nucleus, Which Is An Abnormal Finding Observed In Muscle Biopsies. People With Cnm Begin Experiencing Muscle Weakness At Any Time From Birth To Early Adulthood And Many Patients Die Within The First 18 Months Of Life. Patients Who Survive Longer Require Intense Medical Management And Nearly Uninterrupted Support, Including Permanent Ventilation, Brace With Head Support And Feeding Tubes. The Disease Is Driven By Mutations In Multiple Genes Including Mtm1, Dnm2 And Bin1 And Dynacure Scientists Have Discovered A Link Between An Increase In Dnm2 Protein And The Direct Cause Of The Disease. The Three Classical Forms Of Cnm Are X-Linked Myotubular Myopathy (Xlcnm), Autosomal Dominant Cnm (Adcnm), And Autosomal Recessive Cnm (Arcnm), Which Are All Associated With Poor Prognosis. Myotubular And Centronuclear Myopathies Affect An Estimated 4,000 To 5,000 Patients In The European Union, United States, Japan And Australia.Dyn101, An Investigational Antisense Oligonucleotide Product Candidate Using Ionis Pharmaceuticals' Proprietary Antisense Technology, Is Designed To Reduce The Expression Of Dynamin 2 Protein (Dnm2) For The Treatment Of Myotubular And Centronuclear Myopathies (Cnm).