EMA grants orphan designation to selumetinib for treatment of neurofibromatosis type 1

Aug 3 2018

AstraZeneca and Merck (NYSE:MRK), known as MSD outside the U.S. and Canada, today announced that the European Medicines Agency (EMA) has granted orphan designation to 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 newborns worldwide. The severity of signs and symptoms associated with NF1 can be highly variable, are often mild-to-moderate and may include skin, nerve and skeletal manifestations. Plexiform neurofibromas (PNs) are benign tumors on nerve sheaths that develop in 20-50 percent of patients and, as they continue to increase in number and size, cause moderate-to-severe morbidities such as pain, motor dysfunction and disfigurement.

Sean Bohen, executive vice president, global medicines development and chief medical officer, at AstraZeneca, said, "There is no cure for NF1, a life-long and devastating condition, and current treatment choices for these patients are very limited. The granting of an orphan designation is a positive step forward for children with NF1 and their families."

Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories, said, "NF1 is a relatively rare disease, but can lead to life-threatening complications in those affected by it. This underscores the importance of this collaborative effort between Merck and our partner, AstraZeneca, to help patients impacted by this debilitating genetic condition."

The potential benefit of selumetinib in NF1 is being explored in the Phase 1/2 SPRINT trial in pediatric patients with inoperable NF1-related PNs. Select findings were presented recently at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago by the principal investigators at the National Cancer Institute (NCI). Full results are expected later in 2018.

Orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is seriously debilitating/life threatening and has a prevalence of up to five in 10,000 in the European Union (EU). Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan designation is conferred following a positive opinion by the EMA's Committee for Orphan Medicinal Products. Selumetinib was granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of NF1 in February 2018.