AstraZeneca and Merck announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1).
NF1 is an incurable genetic condition that affects one in 3,000 births with highly-variable symptoms including cutaneous (skin), neurological (nervous system) and orthopedic (skeletal) manifestations. NF1 can cause secondary complications including learning difficulties, visual impairment, pain, disfigurement, twisting and curvature of the spine, high blood pressure and epilepsy. Plexiform neurofibromas (PNs) are tumors that arise from nerve fascicles and tend to grow along the length of the nerve. PNs, a neurological manifestation of NF1, occur in approximately 20-50 percent of NF1 patients causing pain, motor dysfunction and disfigurement.
“Neurofibromatosis type 1 is a devastating condition that can lead to life-threatening complications. There is no known cure for neurofibromatosis and there are limited treatment options to manage symptoms,” Sean Bohen, executive vice president, global medicines development and chief medical officer, AstraZeneca, said.
The potential benefit of selumetinib in NF1 is being explored in the U.S. National Cancer Institute-sponsored phase 1/2 SPRINT trial in pediatric patients with symptomatic NF1-related PNs. Phase II trial results are expected later in 2018.
The FDA’s ODD program provides orphan status to medicines that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S.