In the first quarter, the US Food and Drug Administration (FDA) gave the nod to the first drugs against liver fluke disease and postpartum depression. A new therapeutic modality also entered the pharmacopeia: Sanofi’s camelid-derived nanobody therapy Cablivi. Biogen’s monoclonal antibody (mAb) aducanumab failed in Alzheimer’s disease, surely providing a final nail in the β-amyloid drug target coffin. A modified mRNA therapy for urea cycle disorders and a gene therapy for multiple dystrophy also suffered setbacks. Next quarter, Ardelyx, Sarepta and Clinuvel Pharmaceuticals await franchise-defining FDA decisions.
Drug/company | Indication | FDA approval information |
---|
Cablivi (caplacizumab-yhdp)/Sanofi | Acquired thrombotic thrombocytopenic purpura | 2/6/2019 Recombinant humanized bispecific camelid-derived nanobody against von Willebrand factor |
Esperoct (turoctocog alfa pegol)/Novo Nordisk | Hemophilia A | 2/19/2019 Factor VIII with site-specific glyco-PEGylation within the truncated B-domain |
Herceptin Hylecta (trastuzumab plus hyaluronidase-oysk)/Roche | Breast cancer | 2/27/2019 Humanized IgG1 mAb against HER-2 plus recombinant hyaluronidase, for subcutaneous delivery |
Egaten (triclabendazole)/Novartis | Parasitic and protozoal disease | 2/13/2019 Triclabendazole, a benzimidazole anthelminthic, the first approval for fascioliasis (liver flukes) in the US |
Zulresso (brexanolone)/Sage Therapeutics | Postpartum depression | 3/19/2019 Allopregnanolone, an allosteric small-molecule inhibitor of GABAA receptor, the first approval for postpartum depression |
Sunosi (solriamfetol)/Jazz Pharmaceuticals | Narcolepsy, sleep apnea | 3/20/2019 First small-molecule dual-acting dopamine and noradrenaline uptake inhibitor |
Mayzent (siponimod)/Novartis | Multiple sclerosis | 3/26/2019 Small-molecule oral sphingosine-1-phosphate receptor modulator against lymphocyte trafficking |
This is a preview of subscription content, access via your institution