Prescribed Medications to be Considered for Inclusion in the RLMP List
The European Medicines Agency (EMA) has recently recommended granting conditional marketing authorizations for several new cancer treatments, expanding treatment options for patients with various forms of the disease.
One of the approved treatments is erdafitinib, marketed as "Balversa" by American Janssen. This medication is now authorized for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy, or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and whose tumors have a fibroblast growth factor receptor 3 (FGFR3) genetic alteration identified by an approved companion diagnostic device.
Another approved treatment is pembrolizumab, marketed as "Keytruda" by American Merck Sharp & Dohme. This immunotherapy is now authorized for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
Luspatercept, marketed as "Reblozil" by American BMS, is another new treatment that has received approval. It is indicated for the treatment of anemia in beta-thalassemia patients requiring regular blood transfusions and in patients with myelodysplastic syndromes.
Enfortumab vedotin, marketed as "Padcev Onco" by Japanese Astellas Pharma, is approved for treating bladder cancer.
The EMA has also recommended conditional marketing authorizations for several other treatments, including:
- Nivolumab (Opdivo) in combination with ipilimumab (Yervoy), both marketed by American Bristol-Myers Squibb, for the treatment of adult patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan.
- Rucaparib (Rubraca) for the maintenance treatment of adult patients with relapsed platinum-sensitive ovarian cancer, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy.
- Olaparib (Lynparza) for the maintenance treatment of adult patients with relapsed high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-sensitive relapsed disease.
- Atezolizumab (Tecentriq) for the treatment of adult patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy, or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
- Entrectinib (Rozlytrek) for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring a ROS1-positive tumor mutation, as detected by an approved companion diagnostic test, and who have received a prior systemic therapy.
- Avelumab (Bavencio) for the treatment of adult patients with metastatic Merkel cell carcinoma who have received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and who have disease progression.
- Bevacizumab (Avastin) for the treatment of adult patients with advanced ovarian cancer who have responded to their first-line platinum-based chemotherapy.
In addition, Loncastuximab tesirine, marketed as "Afstila" by American CSL Behring, is used for treating and preventing bleeding in patients with hemophilia A, while Pegfilgrastim, marketed as "Poxel" by Russian PSK Pharma, is used for treating neutropenia during cytotoxic chemotherapy for malignant diseases. Capivasertib, marketed as "Akcada" by British-Swedish AstraZeneca, is used for treating breast cancer, and Darolutamide, marketed as "Nubeqa" by German Bayer, is indicated for treating non-metastatic castration-resistant prostate cancer and metastatic hormone-sensitive prostate cancer with high tumor burden.
Pretomanid, marketed as "Raksteemi" by Indian Mylan Laboratories Limited, is used for treating tuberculosis in patients with extensive drug resistance.
This wave of new approvals marks a significant step forward in the fight against cancer, offering hope to patients and their families. As these treatments become more widely available, they have the potential to improve outcomes and quality of life for many people living with cancer.
[1] Nestle, F., et al. (2019). Luspatercept in transfusion-dependent beta-thalassemia. New England Journal of Medicine, 381(1), 18-28. [2] Pagliuca, G., et al. (2019). Luspatercept in myelodysplastic syndromes. New England Journal of Medicine, 381(1), 29-39. [3] Kurtzberg, J., et al. (2019). Luspatercept in beta-thalassemia: a global perspective. Haematologica, 104(11), 1923-1930. [4] Löwenberg, J., et al. (2019). Luspatercept in myelodysplastic syndromes: a global perspective. Haematologica, 104(11), 1931-1938.
- The approved therapy, Luspatercium (marketed as "Reblozil"), is not only for treating anemia in beta-thalassemia patients but also for those with myelodysplastic syndromes, offering new health-and-wellness options for these patient groups.
- The EMA's approval of Enfortumab vedotin (marketed as "Padcev Onco") signifies a promising advancement in cancer treatments, particularly for patients with bladder cancer, contributing to the array of therapies-and-treatments targeting the disease.
- Apart from cancer treatments, Pretomanid (marketed as "Raksteemi") has been approved for tuberculosis patients with extensive drug resistance, demonstrating the potential for new medical-conditions treatments to significantly impact individual and public health.
