The FDA granted breakthrough status for Merck’s and AstraZeneca’s selumetinib, an experimental drug aimed at a rare genetic pediatric disorder.
Selumetinib is aimed at children three years and older who suffer from neurofibromatosis type 1 (NF1) symptomatic and/or progressive, inoperable plexiform neurofibromas (PN), a rare, incurable genetic disorder that can lead to uncontrolled tumor growth.
The breakthrough therapy designation “acknowledges the significant unmet need of these patients and the potential benefit of selumetinib in this setting,” said Jose Baselga, AstraZeneca’s executive vice president for oncology R&D.
Selumetinib inhibits the MEK enzyme, which researchers hope will stall tumor growth.
The companies are currently testing selumetinib as a monotherapy and in combination with other treatments in a Phase I/II trial.