This marks Yervoy as BMS’ first immuno-oncology medicine approved in Europe for a pediatric indication. The treatment, to be given in a 3 mg/kg dose every three weeks for a total of four administrations, will be marketed in all the European Union’s 28 member states.
Yervoy is a checkpoint inhibitor that binds to the surface molecule cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4), increasing the activity of anti-cancer T-cells, a type of immune cell.
Pediatric melanoma is a rare and severe form of skin cancer, whose patients still need more effective treatments.
“With this approval, we’re able to provide an alternative to young patients whose treatment options have traditionally been limited,” Fouad Namouni, MD, Oncology Development Head at BMS, said in a press release.
The FDA’s decision was based on data from two clinical studies. The first was a Phase 1 trial (NCT01445379) with 33 patients, ages 2 to 21, with relapsed or refractory solid tumors.
Participants received ascending doses of Yervoy – 1, 3, 5, or 10 mg/kg – to determine the best dose with minimum side effects. Treatment was given every three weeks for four doses and then every 12 weeks until disease progression or discontinuation of treatment.
The second study was a Phase 2 trial (NCT01696045) with 12 adolescents, ages 12 to 16, with unresectable stage 3 or 4 malignant melanoma.
Participants received one of two Yervoy doses – 3 mg/kg in four patients, and 10 mg/kg in eight patients – every three weeks for four doses.
Among patients aged 12 and older treated across both studies, two achieved an effective tumor response to the treatment, including one patient with a partial response that lasted for 16 months.
The benefits of Yervoy in patients ages 12 and older are also backed by pharmacological data in adults showing that the 3 mg/kg dose of Yervoy in the pediatric and adult groups is comparable. These two age groups share similarities in tumor biology and progression of advanced melanoma, which enables extrapolation of results from one group to another.
Yervoy is now approved in the E.U. for pediatric patients with advanced melanoma for 90 minutes of intravenous administration of 3 mg/kg every three weeks for a total of four administrations.
The U.S. Food and Drug Administration approved Yervoy for pediatric advanced melanoma in July 2017.