Table 4.
Promising Late-Phase Drugs for Various Patient Populations and Tumor Types
| Drug trade name (generic) | Manufacturer | Expected indication/therapeutic class/route | Development stage/comments (expected approval) |
|---|---|---|---|
| Rolapitant | Tesaro | For the prevention of chemotherapy-induced nausea and vomiting; selective NK-1 receptor agonist; oral | NDA: 9/2014 Est. PDUFA: 9/5/15 IV formulation in phase 3 trials |
| Yondelis (trabectedin) | Janssen Pharmaceuticals | For advanced soft-tissue sarcoma, including liposarcoma and leiomyosarcoma; multimodal therapy; IV infusion | NDA: 11/25/14 Priority review: 2/3/15 Est. PDUFA: 11/24/15 |
| TAS-102 (trifluridine + tipiracil hydrochloride) | Taiho Oncology | For third- or fourth-line treatment of refractory metastatic colorectal cancer; combination of antineoplastic nucleoside analog and a hydrochloride | NDA: 2/23/15 PDUFA: 12/19/15 |
| Rindopepimut | Celldex Therapeutics | Immunotherapy for EGFRvIII-positive glioblastoma; intradermal | BT for glioblastoma: 1/23/15 Phase 3 trials |
| MM-398 (irinotecan liposome) | Merrimack Pharmaceuticals | For second-line treatment of metastatic pancreatic cancer; nanotherapeutic derivative of irinotecan; IV | NDA: 4/27/15 Fast track: 11/19/14 |
| Sonidegib (LDE225) | Novartis | For advanced basal-cell carcinoma; selective smoothened inhibitor; oral | NDA: 10/2014 Est. approval: 9/2015 |
| PLX3397 | Plexxikon | Recurrent glioblastoma; also in combination with pembrolizumab for advanced melanoma and multiple other solid tumors; tyrosine kinase inhibitor; oral | Phase 2 trials |
| Tivantinib | ArQule | Treatment of c-MET diagnostic-high inoperable hepatocellular carcinoma treated with 1 previous sorafenib therapy; c-MET inhibitor; oral | Phase 3 trials |
| Niraparib | Tesaro | For ovarian cancer; PARP inhibitor; oral | Phase 3 trials |
| Tasquinimod | Active Biotech Research | For castration-resistant prostate cancer; allosteric modulator of HDAC4; oral | Phase 3 trials |
| Algenpantucel-L | NewLink Genetics | Immunotherapy vaccine for resectable or locally advanced unresectable pancreatic cancer; intradermal injection | Phase 3 trials |
BT indicates breakthrough therapy; EGFR, epidermal growth factor receptor; Est., estimated; HDAC, histone deacetylase; IV, intravenous; NDA, New Drug Application (submitted); NK, neurokinin; PARP, poly (ADP-ribose) polymerase; PDUFA, Prescription Drug User Fee Act.