The long-awaited thumbs-up for a gene therapy finally came through, with the November approval by the EMA of Glybera (alipogene tiparvovec) for patients lacking lipoprotein lipase. Other notable approvals are Aubagio (teraflunomide), the second small-molecule drug for multiple sclerosis, and Kyprolis (carfilzomib), an irreversible proteasome inhibitor for multiple myeloma. Some drugs addressing unmet oncology needs (pancreatic and mesothelioma) are showing positive signs in trials, and the first systemic antisense drug, Kynamro (mipomersen sodium), will be going before the FDA shortly.
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DeFrancesco, L. Drug pipeline: Q312. Nat Biotechnol 30, 1164 (2012). https://doi.org/10.1038/nbt.2446