In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis Institutes for BioMedical Research, http://rarediseases.org/rarediseases/spinal-muscular-atrophy/, https://www.hrsa.gov/advisory-committees/heritable-disorders/rusp/index.html. J Neurol. Pour son premier trimestre plein de commercialisation aux Etats-Unis, le Zolgensma a généré 160 millions de dollars entre juin et fin septembre. Monitor platelet counts before Zolgensma infusion and on a regular basis afterwards. Who would get Regeneron's COVID-19 antibody treatment? The free newsletter covering the top industry headlines, By signing up to receive our newsletter, you agree to our, Press release from RCPE - Research Center Pharmaceutical Engineering GmbH, From no drugs to 3: Patients with spinal muscular atrophy now face hard choices. About Novartis Novartis is reimagining medicine to improve and extend people's lives. Neurology. Without a functional SMN1 gene, infants with SMA lose the motor neurons responsible for muscle functions such as breathing, swallowing, speaking and walking. This level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families battling SMA Type 1. Yahoo forma parte de Verizon Media. Basel, May 24, 2019 - AveXis, a Novartis company, today announced the US Food and Drug Administration (FDA) has approved Zolgensma ® (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Puedes cambiar tus opciones en cualquier momento visitando Tus controles de privacidad. Comparison of the results of the low-dose cohort to the results of the high-dose cohort shows a dose-response relationship that supports the effectiveness of Zolgensma. La FDA compte porter l’affaire en justice car Novartis a fait part du problème après avoir obtenu l’approbation de l’agence. ZURICH — Novartis’s bid to expand its $2.1 million-per-patient gene therapy Zolgensma to more spinal muscular atrophy patients faces a possible delay after U.S. regulators requested another study in older children getting the drug via a spinal infusion. Patients experienced onset of clinical symptoms consistent with SMA before 6 months of age. Still, an analyst said the FDA’s request for more data – from children aged 2 to 5 years getting Zolgensma via spinal infusion – would delay Novartis’s efforts to expand approval to at least late 2023, damaging financial prospects especially after Roche’s rival oral medicine Evrysdi hit the market in August. Spinal Muscular Atrophy. About Spinal Muscular Atrophy (SMA) SMA is a severe neuromuscular disease characterized by the loss of motor neurons leading to progressive muscle weakness and paralysis. Dans le cadre de ce contrat,[…], Pierre Fabre va investir 4,5 M€ pour relocaliser à Gaillac des actifs produits en Allemagne, Covid-19 : Le Royaume-Uni débute l’évaluation du vaccin d’AstraZeneca, Covid-19 : La biotech montpelliéraine MedinCell avance sur son évaluation de l’ivermectine en traitement préventif, Coronavirus : Tout comprendre sur les différents vaccins en développement, Covid-19 : Moderna va pouvoir déposer une demande d'AMM en Europe pour son vaccin.