Type 1 Spinal Muscular Atrophy (SMA)

Novartis says data confirms benefit of Zolgensma gene therapy for babies

Babies, Drug Pricing, Efficacy, FDA/Regulatory, Gene Therapy, Interim Data, Rare Genetic Diseases, Spinal Muscular Atrophy (SMA), Studies, Therapeutics, Type 1 Spinal Muscular Atrophy (SMA)

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.

Novartis wins key European recommendation for gene therapy Zolgensma

Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (EMA), FDA/Regulatory, Gene Therapy, Recommended For Approval, Type 1 Spinal Muscular Atrophy (SMA)

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 on Track for Approval for Spinal Muscular Atrophy

Analysts, 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).