Novartis said new interim data from an ongoing phase clinical trial for the company’s $2.1 million-per-patient gene therapy Zolgensma showed spinal muscular atrophy patients experienced significant therapeutic benefit.
Swiss drugmaker Novartis won a key European recommendation for the company’s gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.
Genentech’s Risdiplam showed significant improvement in motor function in people aged 2-25 who have been diagnosed with Type 2 or 3 spinal muscular atrophy.
Genentech’s Risdiplam on Track for Approval for Spinal Muscular AtrophyAnalysts, Bi-allelic Mutations, Clinical Trials, Drug Pricing, FDA, FDA/Regulatory, Infants, Neuromuscular diseases, New Drug Application (NDA), Potential Blockbusters, R&D, Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy (SMA), Survival motor neuron 1 (SMN1) gene, Type 1 Spinal Muscular Atrophy (SMA)
Roche company Genentech announced positive top-line results from the pivotal Part 2 of the FIREFISH study looking at risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA).
England’s National Health Service reached a deal to pay for U.S. drugmaker Biogen Inc.’s expensive spinal muscle atrophy treatment Spinraza.
PTC Therapeutics announced interim data from part 1 of the company’s open-label clinical trials of risdiplam (RG7916) for Type 1, 2 and 3 spinal muscular atrophy (SMA).
South San Francisco-based Cytokinetics released data from the company’s Phase II clinical trial of reldesemtiv in patients with spinal muscular atrophy at the 2018 Annual Cure SMA Conference held in Dallas.