At the end of May, Zolgensma was approved by the US Food and Drug Administration (FDA) as a gene therapy for children up to the age of two with spinal muscular atrophy (SMA). This was followed on June 3 by the European Commission’s marketing authorization for Zynteglo, a gene therapy against transfusiondependent β-thalassemia in patients 12 years or older. While patient groups hailed the news, the price of these products provoked sticker shock: Zolgensma costs $2.1 million; Para acessar a publicação na íntegra clique aqui Fonte: Nature Biotechnology…
FDA new drug approvals in Q2 2019
The most prominent approval in the second quarter of 2019 was Novartis’s pioneering gene therapy for spinal muscular atrophy (SMA), Zolgensma (onasemnogene abeparvovec), a rare neuromuscular disease with a typical life expectancy of less than 2 years if untreated. Para acessar a publicação na íntegra clique aqui Fonte:NATure Reviews | Drug DIScovery volume 18 | August 2019 | 575…