The US Food and Drug Administration (FDA) continues to approve products at a rapid clip, with 17 new molecular entity (NME) and biologic approvals in the quarter. Over 40 years after the first monoclonal antibody (mAb) was approved—muromonab (Ortho Kung/OKT3), an anti-CD3 murine mAb for use in graft-versus-host disease—FDA will consider a humanized version of the drug for type 1 diabetes. Elsewhere, CRISPR Therapeutics published promising one-year results on its CRISPR–Cas9 therapy against the BCL111A erythroid-specific enhancer in transfusion-dependent β-thalassemia and sickle cell disease. Bristol Myers Squibb (BMS) and bluebird bio’s Abecma (idecabtagene vicleucel) became the first chimeric antigen receptor (CAR)-T cell therapy to be approved for multiple myeloma and the first directed against an antigen other than CD19. Regeneron’s Evkeeza (evinacumab-dgnb) is the first mAb approved for familial hypercholesterolemia that targets the angiopoietin-like 3 protein. A double dose of bad news came in Huntington’s disease, with clinical setbacks for two antisense oligonucleotide (ASO) therapies: Ionis/Roche’s tominersen and Wave’s WVE-120102. Lilly reported positive trial results for its Alzheimer’s disease therapy mAb donanemab; by next quarter’s end, Biogen might finally get an answer from regulators on its controversial anti–Alzheimer’s disease mAb, aducanumab.
Drug/company | Indication | Drug information |
|---|
Lupkynis (voclosporin)/Aurinia | Lupus nephritis | 1/22/2021 FDA approved this small-molecule calcineurin phosphatase inhibitor, the first oral drug for lupus nephritis |
Breyanzi (lisocabtagene maraleucel)/BMS | Diffuse large B cell lymphoma | 2/5/2021 FDA approved autologous CAR-T cells, expanded in culture, expressing CD19 and a truncated epidermal growth factor receptor (EGFR) |
Ukoniq* (umbralisib)/TG Therapeutics | Marginal zone lymphoma; indolent non-Hodgkin’s lymphoma | 2/5/2021 FDA granted accelerated approval to this small molecule targeting phosphatidylinositol 3-OH kinase (PI3K)-δ and casein kinase 1 (CK1)-ε. |
Evkeeza (evinacumab-dgnb)/Regeneron | Dyslipidemia/hypercholesterolemia | 2/11/2021 FDA approved this fully human IgG4 mAb against angiopoietin-like 3 that has a stabilizing mutation in the hinge. |
Amondys 45* (casimersen)/Sarepta Therapeutics | Duchenne muscular dystrophy | 2/25/2021 FDA gave accelerated approval to this 22-residue exon-45-skipping phosophorodiamidate ASO. |
Nulibry (fosdenopterin)/BridegeBio Pharma | Molybdenum cofactor deficiency | 2/28/2021 FDA approved cyclic pyranopterin monophosphate, an intermediate in the synthesis of molybdenum cofactor |
Abecma (idecabtagene vicleucel/BMS | Multiple myeloma | 3/29.2021 FDA approved this CAR-T cell therapy with anti-B-cell maturation antigen (BCMA) single-chain variable fragment (scFv) fused to the CD137 (4-1BB) co-stimulatory and CD3ζ signaling domains |
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