The US Food and Drug Administration (FDA) picked up the pace of drug registrations after a sluggish first quarter. Many were accelerated approvals for cancer indications, including two small molecules — Incyte’s Pemazyre and Novartis’s Tabrecta — approved with companion diagnostics from Foundation Medicine. The agency halted two adeno-associated virus (AAV) gene therapy trials, from Audentes and Sarepta, for safety reasons; next quarter, the European Medicines Agency (EMA) will consider AAV gene therapies from Biomarin and PTC Therapeutics for hemophilia and aromatic l-amino acid decarboxylase (AADC) deficiency, respectively. A small interfering RNA (siRNA) oligonucleotide therapy for hypercholesterolemia from Novartis (The Medicines Company) also will be under evaluation. Several companies reported clinical results from drugs repurposed for COVID-19, with Gilead and Kiniksa reporting preliminary data showing improvements in duration of illness and certain outcomes for Veklury (remdesivir) and mavrilimumab, respectively.
Drug/company | Indication | Drug information |
|---|
Enspryng (satralizumab)/Roche | Neuromyelitis optica (Devic’s syndrome) | 4/24/2020 A double-blind, placebo-controlled trial of this humanized IgG2 mAb against IL-6 receptor reduced the risk of relapse by 55% (Lancet Neurol. 19, 402–412, 2020) |
Veklury (remdesivir)/Gilead | COVID-19 | 5/22/2020 A double-blind, randomized, placebo-controlled trial of this small-molecule nucleotide analog viral RNA-dependent RNA polymerase inhibitor reduced recovery to 11 days, as compared with 15 days for placebo (New Engl. J. Med. https://doi.org/10.1056/NEJMoa2007764, 2020) |
Mavrilimumab/Kiniksa | COVID-19 | 6/16/2020 In an open-label clinical trial with this fully human IgG4 mAb against granulocyte macrophage colony stimulating factor receptor alpha in patients with severe disease, none of 13 patients treated with drug died while 7 (27%) in the control group died. (Lancet Rheumatol. https://doi.org/10.1016/S2665-9913(20)30170-3, 2020) |
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