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Mês: agosto 2018

US approves first RNA interference drug

The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food and Drug Administration (FDA). Alnylam Pharmaceuticals got the green light for Onpattro (patisiran), to treat hereditary transthyretin amyloidosis – a rare, progressive and often fatal disease. A mutation in patients with this hereditary disease makes a toxic form of transthyretin protein that is deposited in the heart and peripheral nerves. Patisiran silences this gene using short pieces of double-stranded RNA that target and…

FDA new drug approvals in Q2 2018

The second quarter of 2018 saw an uptick in drug approvals as 15 new drugs received a green light from the FDA (TABLE 1), compared with 6 in the first quarter. Among the most notable of the new crop was GW Pharmaceuticals’ cannabidiol (Epidiolex), the first cannabis-derived drug to gain FDA approval. Epidiolex was cleared to treat two very rare types of severe childhood epilepsy, Lennox–Gastaut syndrome and Dravet syndrome, and is forecast to reach blockbuster sales of US$2.35 billion by 2024. However, it is hard to know how much of a threat…