FDA Approvals Boost Competition in Treatment Landscapes

Therapeutic developments in cancer were rewarded with a wave of approvals in 2019. Tumor types such as breast cancer and prostate cancer saw multiple approvals, while continued research offered more targeted therapies across cancer types. May was the busiest month, with 8 new indications and the novel drug approval of alpelisib (Piqray) in combination with fulvestrant for hormone receptor—positive, HER2-negative, PIK3CA-mutated metastatic breast cancer.

In all, innovations in oncology care resulted in FDA approval for 12 novel agents across hematology/ oncology settings.

One such novel agent, entrectinib (Rozlytrek) was approved for the treatment of multiple tumor types in patients harboring NTRK gene fusions. This is the third drug in 3 years to be approved based on a biomarker rather than a specific anatomical location, following pembrolizumab (Keytruda) for microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) tumors in 2017 and larotrectinib (Vitrakvi) for NTRK-positive tumors in 2018. Entrectinib was simultaneously approved for adults with non—small cell lung cancer (NSCLC) whose tumors are ROS1 positive.

Bladder cancer gained 2 new therapies for previously treated patients with urothelial carcinoma: enfortumab vedotin-ejfv (Padcev) and erdafitinib (Balversa).

Table. The Year in Innovation (Click to Enlarge)

Luspatercept-aamt (Reblozyl) shook up the hematology space in November with an approval for the treatment of anemia in adult patients with ß-thalassemia who require red blood cell transfusions.

Elsewhere in the treatment landscape, there were several approvals for drugs to be used earlier for earlier stages of prostate cancer: enzalutamide (Xtandi) and apalutamide (Erleada) for metastatic castration-sensitive disease and darolutamide (Nubeqa) for nonmetastatic castration-resistant tumors. The FDA has now approved 3 drugs in this space based on metastasis-free survival.

Immunotherapies aimed at the PD-1/PD-L1 pathway continued to gather new indications. These included an approval for pembrolizumab in combination with lenvatinib (Lenvima) for patients with endometrial carcinoma that is not MSI-H or dMMR and who have disease progression and are not candidates for curative surgery or radiation following systemic therapy.

The biosimilar sector for oncology drugs also saw an increase in the number of approved trastuzumab biosimilars, with 3 new competitors to the reference product. Elsewhere in the biosimilar space, the FDA greenlit the third biosimilar for pegfilgrastim and the second each for rituximab and bevacizumab.

Table. The Year in Innovation (Click to Enlarge)

KEY

AML indicates acute myeloid leukemia; aTTP, acquired thrombotic thrombocytopenic purpura; CLL, chronic lymphocytic leukemia; CRC, colorectal cancer; CRPC, castration-resistant prostate cancer; DLBCL, diffuse large B-cell lymphoma; ES-SCLC, extended-stage small cell lung cancer; FL, follicular lymphoma; gBRCAm, germline BRCA-mutant; GEJ, gastric esophagogastric junction; GVHD, graft-versus-host disease; HCC, hepatocellular carcinoma; HRD, homologous recombination deficient; MCL, mantle cell lymphoma; mCSPC, metastatic castration-sensitive prostate cancer; MM, multiple myeloma; MZL, marginal zone lymphoma; NHL, non-Hodgkin lymphoma; NSCLC, non—small cell lung cancer; RCC, renal cell carcinoma; SLCL, small cell lung cancer; SLL, small lymphocytic lymphoma; TNBC, triple-negative breast cancer; VTE, venous thromboembolism.

For more approval information visit, bit.ly/35zei0t.

To close out the year, the FDA approved a new antibody-drug conjugate, fam-trastuzumab deruxtecan- nxki (Enhertu), as a third-line treatment for patients with unresectable or metastatic HER2- positive breast cancer and expanded the indication for olaparib (Lynparza), a PARP inhibitor, in the maintenance setting for BRCA-positive pancreatic cancer.