US FDA approval tracker: November 2019

November was a remarkable month for speedy approvals: the FDA green-lit five novel medicines ahead of their respective PDUFA dates. Sickle cell saw two new arrivals: Novartis’s Adakveo, for the prevention of the painful vaso-occlusive crises associated with the disorder, and Global Blood’s Oxbryta, which can boost haemoglobin levels. The latter only has conditional approval, with a confirmatory trial needed to answer questions about its impact on longer term outcomes. Meanwhile Alnylam gained approval of its second RNAi drug, Givlaari, and Beigene received its first ever US approval with the BTK inhibitor Brukinsa. Brukinsa can be used as a second-line treatment for mantle cell lymphoma; it is also being tested in the much larger indication of chronic lymphocytic leukaemia with initial cohort data from the phase III Sequoia trial due at Ash. At the other end of the spectrum is Lipocine, which received a third complete response letter for its hypogonadism treatment Tlando; shares crashed 71% on the news. Meanwhile Agile, which has also received two CRLs for its contraceptive patch Twirla, will have to wait a little longer: the projects PDUFA date has been delayed until February.