Novartis’s breast cancer drug Piqray (alpelisib) was the first new drug application for a new molecular entity approved under the US Food and Drug Administration (FDA) Real-Time Oncology Review (RTOR) pilot program, which aims to speed approvals while ensuring safety. The first targeted small-molecule kinase inhibitor against bladder cancer was approved with an accompanying companion diagnostic. A second drug with marginal efficacy in female sexual arousal disorder, the peptide Vyleesi, was approved (following Addyi in 2016). Phase 1/2 trials showed that an antisense oligonucleotide and an ex vivo lentiviral gene therapy produced responses in Huntington’s disease and Wiskott–Aldrich syndrome, respectively. Two new migraine drugs are in queue for decisions in the fourth quarter.
Drug/company | Indication | Drug information |
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Luspatercept/Acceleron Pharma | Thalassemia | 12/4/2019 FDA PDUFA date for modified human activin receptor type-IIb extracellular domain linked to a human IgG1 Fc domain, which modulates growth and differentiation factor (GDF) ligands and receptors and selectively activates late stages of erythropoiesis by inhibiting transforming growth factor-β |
Ubrogepant/Allergan | Migraine and other headaches | 12/15/2019 FDA PDUFA date for this small-molecule oxazolidinone-based calcitonin gene-related peptide (CGRP) receptor antagonist |
Lasmiditan/Eli Lilly | Migraine and other headaches | 11/14/2019 FDA PDUFA date for small molecule targeting 5-hydroxytryptamine 1F (HT1F) receptors expressed in the trigeminal nerve pathway, without vasoconstriction |
RVT-802/Enzyvant Therapeutics | Primary immunodeficiencies | 11/22/2019 FDA PDUFA date for allogeneic thymic tissue transplant for restoring T-cell development in absence of thymus |
ATIR101/Kiadis Pharma | Bone marrow transplant | 9/30/2019 EMA decision on allogeneic CD34+ T cells depleted of actively dividing cells ex vivo via treatment with photosensitizer 4,5-dibromorhodamine methyl ester (TH9402) |
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