The drugs are enfortumab vedotin (Padcev, Astellas/Seagen) for urothelial cancer, tepotinib (Tepmetko, Merck) for non-small cell lung cancer (NSCLC), and voxelotor (Oxbryta, Global Blood Therapeutics) for sickle cell hemolytic anemia.
EMA’s Committee for Medicinal Products for Human Use gave the nod for marketing authorization on December 16, and agency approval generally follows about 6 weeks later. All three products are already approved in the United States.
Enfortumab Vedotin for Urothelial Cancer
The recommendation for Astellas’ antibody-drug conjugate infusion was based on the phase 3 EV-301 trial, which found about a 4-month median overall survival benefit with the antibody-drug conjugate versus investigator-chosen chemotherapy across 608 patients with locally advanced or metastatic urothelial carcinoma previously treated with platinum-based chemotherapy and a PD-1 or PD-L1 inhibitor.
The planned European indication is for adults with locally advanced or metastatic disease who met the same criteria — previous platinum-based chemotherapy plus a PD-1 or PD-L1 inhibitor.
The US Food and Drug Administration (FDA) approval came in December 2019, and with the same indication as well as for patients ineligible for cisplatin-containing chemotherapy who have had one or more prior lines of therapy. The US labeling carries a boxed warning of severe and fatal skin reactions including Stevens-Johnson syndrome and toxic epidermal necrolysis.
Tepotinib for NSCLC
The recommendation for Merck’s tepotinib, a once-daily oral MET inhibitor, followed results from the phase 2 VISION study. The study found an investigator-assessed response rate of 56% across 152 patients with advanced or metastatic NSCLC with a confirmed MET exon 14 skipping mutation, regardless of previous therapy.
The planned European indication will be for monotherapy in adults with advanced disease harboring the mutation who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.
Voxelotor for Sickle Cell Disease
Voxelotor is an oral hemoglobin S polymerization inhibitor from Global Blood Therapeutics.
The European approval recommendation was based on a phase 3 trial in 274 patients with sickle cell disease that found a greater than 1 g/dL increase in hemoglobin levels at 24 weeks in 51.1% of patients, versus 6.5% randomized to placebo, regardless of whether patients were on concomitant hydroxyurea.
The small molecule binds and stabilizes hemoglobin, preventing the hemoglobin polymerization that causes red blood cells to sickle.
“There is a high unmet need for medicines to treat hemolytic anemia” in sickle cell disease because available treatment options are limited to blood transfusions and allogenic hematopoietic stem cell transplantation,” EMA explained in a press release announcing the approval recommendation.
The planned European indication is for treating hemolytic anemia in sickle cell disease in patients 12 years or older either alone or in combination with hydroxycarbamide (hydroxyurea).
M. Alexander Otto is a physician assistant with a master’ s degree in medical science, and an award-winning medical journalist who worked for several major news outlets before joining Medscape. He is an MIT Knight Science Journalism fellow. Email: [email protected]
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