| Company | Product | Description | Indication | Status |
| Brainstorm Cell Therapeutics Inc., of New York | Nurown | Autologous mesenchymal stem cell-neurotrophic factor-producing cell therapy | Amyotrophic lateral sclerosis | Representatives from FDA's CBER confirmed during planned meeting that fully enrolled trial is collecting relevant data to assess efficacy; agency agreed to work collaboratively to identify regulatory pathway, including opportunities to expedite statistical review of phase III data |
| Eli Lilly Canada, a unit of Eli Lilly and Co., of Indianapolis | Taltz (ixekizumab) | IL-17 antagonist; interleukin 17A ligand inhibitor | Ankylosing spondylitis | Health Canada granted approval for individuals who responded inadequately or are intolerant to conventional therapy |
| Fennec Pharmaceuticals Inc., of Research Triangle Park, N.C. | Pedmark | Sodium thiosulfate | Prevention of cisplatin-induced hearing loss (ototoxicity) in children with solid tumors | Completed rolling submission of an NDA to the FDA for intravenous use; also submitted an MAA to the EMA |
| Incyte Corp., of Wilmington, Del. | Capmatinib | Selective MET inhibitor | Non-small-cell lung cancer | FDA accepted the NDA and granted priority review; seeking approval for use in locally advanced or metastatic MET exon 14-skipping mutated disease |
| Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | ION-373 | Designed to stop a mutated gene from producing excess glial fibrillary acidic protein | Alexander disease | EMA granted orphan drug designation |
| Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Darzalex | Daratumumab | Relapsed/refractory multiple myeloma | Submitted an sBLA to the FDA for Darzalex in combination with Kyprolis (carfilzomib) and dexamethasone |
| Logicbio Therapeutics Inc., of Cambridge, Mass. | LB-001 | Recombinant adeno-associated vector with human methylmalonyl-COA mutase gene | Methylmalonic acidemia | The FDA placed a clinical hold on the IND submission |
| Lyndra Therapeutics Inc., of Watertown, Mass. | Ultra-long-acting oral therapy | Designed to open inside the stomach and deliver drugs over time | Schizophrenia | Filed an IND with the FDA |
| Marker Therapeutics Inc., of Houston | MultiTAA | MAPP multi-antigen targeted T-cell therapy | Post-transplant acute myeloid leukemia | FDA lifted the clinical hold on planned trial investigating safety and efficacy |
| Novartis AG, of Basel, Switzerland | Capmatinib (INC-280) | MET inhibitor | METex14 mutated advanced non-small-cell lung cancer | FDA granted priority review |
| Phasebio Pharmaceuticals Inc., of Malvern, Pa. | PB-2452 | Monoclonal antibody antigen-binding fragment targeting ticagrelor | Antiplatelet reversal | Granted PRIME designation by the EMA |
| Sirnaomics Inc., of Gaithersburg, Md. | STP-705 | siRNA therapeutic | Hepatocellular carcinoma | Granted orphan drug designation by the FDA |
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Notes For more information about individual companies and/or products, see Cortellis. |