The third quarter saw US Food and Drug Administration approvals for two anti-infectives. Pretomanid, indicated for drug-resistant tuberculosis (TB), is only the third TB drug approved over the past 50 years and the first antibiotic to be developed by a non-profit, the TB Alliance. Elsewhere, MSD Merck got the green light for Recarbrio, which is approved for intra-abdominal and for urinary and reproductive tract infections. Two new cancer drugs were approved through the agency’s accelerated approval pathway: Karyopharm’s Xpovio and Roche/Genentech’s Rozlytrek. In early 2020, the FDA will review two sickle cell anemia drugs working through novel mechanisms. Meanwhile, Sarepta’s second exon-skipping antisense drug for muscular dystrophy got pushback from the regulator; its predecessor, Exondys 51, was approved in 2016.
Drug/company | Indication | Drug information |
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Roxadustat/AstraZeneca | Anemia | 7/24/2019 In phase 3 double-blind, placebo-controlled trial conducted at 27 sites in China, this small-molecule second-generation hypoxia-inducible factor prolyl hydroxylase inhibitor produced a statistically significant increase in hemoglobin for 26 weeks (N. Engl. J. Med. 381, 1001–1010, 2019) |
Waylivra (volanesorsen)/Akcea Therapeutics | Dyslipidemia / hypercholesterolemia | 8/8/2019 In a phase 3 double-blind, randomized, placebo-controlled trial of this 2′-O-methoxyethyl antisense oligonucleotide that targets apolipoprotein CIII mRNA, patients with familial chylomicronemia syndrome saw a statistically significant mean reduction in triglycerides of 77% from baseline at three months whereas patients on placebo had an 18% increase (N. Engl. J. Med. 381, 531–542, 2019) |
Inebilizumab/Viela Bio | Neuromyelitis optica (NMO or Devic’s syndrome) | 9/6/2019 In a randomized, placebo-controlled, double-blind phase 2/3 trial, this humanized, Fc-afucosylated IgG1 mAb against CD19 reduced attacks, risk of disability worsening, hospitalizations, and new magnetic resonance imaging lesions in NMO compared with placebo (Lancet 394, 1352–1363, 2019) |
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