On 14 September 2017, the European Medicines Agency (EMA) issued positive opinions for the approval of four new treatment options for oncology and hematology, including a biosimilar of trastuzumab for breast cancer, padeliporfin for vascular-targeted photodynamic therapy (VTP) of low-risk prostate cancer, maintenance niraparib for platinum-sensitive relapsed ovarian cancer, and nilotinib for pediatric patients.
- Green Light for First Trastuzumab Biosimilar. Ontruzant® (Samsung Bioepis UK Limited), a biosimilar of trastuzumab, was recommended for the treatment of patients with human epidermal growth factor receptor 2 (HER2)-positive early and metastatic breast cancer and HER2-positive metastatic gastric cancer. This is the first biosimilar of trastuzumab to be approved in Europe. It has been shown to be highly similar to its reference product, Herceptin®, in a series of quality, safety, and efficacy studies, including a phase I pharmacokinetics study in healthy volunteers and a phase III trial in patients with early or locally advanced HER2-positive breast cancer.
- New Focal Therapy for Localized Prostate Cancer. The EMA recommended approval for padeliporfin (Tookad®, STEBA Biotech), a sensitizer used as part of focal vascular-targeted photodynamic therapy (VTP), for patients with previously untreated, unilateral, low-risk prostate cancer. This recommendation was based on results from a phase III trial in which padeliporfin VTP resulted in a doubling in time to progression compared to active surveillance (28.3 months vs 14.1 months; HR 0.34, P<.0001) in patients with low-risk, previously untreated prostate cancer.
- PARP Inhibitor Recommended for Maintenance Therapy in Ovarian Cancer. The EMA recommended approval of niraparib (Zejula®, TESARO UK Limited), a poly(ADP-ribose) polymerase (PARP) 1/2 inhibitor, for maintenance therapy in patients with platinum-sensitive, relapsed high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who following platinum-based chemotherapy. This decision was based on results from the randomized, phase III NOVA trial, in which niraparib maintenance was shown to significantly lengthen progression-free survival compared to placebo in patients regardless of BRCA mutation or homologous recombination deficiency status. Niraparib has been available in the US since March 2017.
- Nilotinib Indication Expanded to Include Pediatric CML. The EMA recommended expanding the indication of nilotinib (Tasigna®, Novartis) to include pediatric patients with newly diagnosed, Philadelphia chromosome (Ph)-positive chronic-phase chronic myelogenous leukemia (CML), and pediatric patients with Ph-positive chronic-phase CML who are resistant or intolerant to imatinib.