Yervoy (ipilimumab) has received a positive review by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP), recommending its approval for the treatment of unresectable or metastatic melanoma in patients age 12 and older.
The CHMP’s recommendation will now be reviewed by the European Commission, the authority that approves medicines for the European Union.
“Pediatric melanoma is a particularly rare cancer, with limited treatment options for children in the E.U. impacted by the disease,” Murdo Gordon, executive vice president and chief commercial officer at Bristol-Myers Squibb, said in a press release.
“We are pleased with today’s positive CHMP opinion and look forward to hearing from the E.C., as we continue to develop and deliver new therapies for the pediatric cancer community,” he added.
Yervoy is an immune checkpoint inhibitor that targets the surface molecule CTLA-4, boosting the activity of anti-cancer T-cells. A broad clinical development program is currently evaluating its potential to treat several types of cancers.
In 2011, Yervoy was approved by the U.S. Food and Drug Administration (FDA) and the EMA as a stand-alone therapy for the treatment of adult patients with unresectable or metastatic melanoma. The treatment was approved as a 3 mg/kg dose, given every three weeks, for a total of four doses.
While the treatment was approved for adults, the tumor biology of advanced melanoma is very similar between adults and children, suggesting that Yervoy would also work in younger patients.
Indeed, clinical data from 45 pediatric patients revealed the drug was effective in this patient group. The Phase 1 trial (NCT01445379) included 33 patients ages 2 to 21 with relapsed or refractory solid tumors. The Phase 2 trial (NCT01696045) enrolled patients ages 12 to 16 with unresectable advanced melanoma.
Across both studies, a total of 17 melanoma patients ages 12 or older were treated with Yervoy. Two patients experienced effective tumor response to the treatment, including one patient with a partial response that lasted for 16 months.
The findings, along with extensive data collected from adult patients demonstrating the safety and effectiveness of Yervoy, supported the CHMP’s positive recommendation. They also led the U.S. Food and Drug Administration (FDA) to approve Yervoy for pediatric patients with advanced melanoma in July 2017.