Cure SMA Announces $5 Million in New Research, Care Funding

Cure SMA recently announced it has committed $5 million in new funding to advance research and care strategies for spinal muscular atrophy (SMA) over the next 12 months. A bit more than half of the total funding will go directly to support local care. The remainder will fund basic…

Potential SMA Therapy, RG7916, for Types 2 and 3 Advancing in Clinical Trial in Europe

A Phase 2 clinical trial evaluating the efficacy and safety of RG7916 in children and adults with type 2 or 3 spinal muscular atrophy has advanced into a second and possibly pivotal phase. The study is part of a development program jointly led by PTC Therapeutics, Roche and the SMA Foundation. An interim analysis from the trial's first part demonstrated an exposure-dependent increase in the SMN protein, which is deficient in SMA patients. RG7916 continues to be well-tolerated at all doses and no drug-related safety findings led to any patients withdrawing from part one. RG7916 is drug that can be taken by mouth that impacts SMN2. Because SMA is caused by a defect in the SMN1 gene, the SMN2 gene has been explored as a potential replacement to guarantee the production of the SMN protein. RG7916 is also being investigated in babies with type 1 or infant-onset SMA in a Phase 2 trial called FIREFISH. This study, running at sites in the U.S. and Europe, is currently recruiting infants ages 1 to 7 months old. PTC Therapeutics and the SMA Foundation initially began working on this potential therapy in 2006, and Roche began to participate in 2011, when it acquired an exclusive worldwide license to this splicing program.  The U.S. Food and Drug Administration (FDA) designated RG7916 an orphan drug for the treatment of SMA in January 2017.

#NORDSummit – FDA Approach to Approvals, Therapy Costs and ‘Right to Try’ Among Issues at NORD Summit

Parents and caregivers of patients with spinal muscular atrophy (SMA) take great interest in orphan drug development, soaring healthcare costs, patient assistance programs and controversial “right-to-try” legislation — all issues on the agenda during the upcoming Rare Disease & Orphan Products Breakthrough Summit. “One of the chief challenges of 21st-century…