The European Medicines Agency (EMA) has granted orphan designation to selumetinib, a MEK 1/2 inhibitor which is being developed by AstraZeneca and Merck to treat neurofibromatosis type 1 (NF1).
AstraZeneca licensed selumetinib from Array BioPharma in 2003. AstraZeneca and Merck have entered a co-development and co-commercialisation agreement for selumetinib in 2017.
NF1 is an incurable genetic condition, which is said to affect one in 3,000 newborns across the globe.
Plexiform neurofibromas (PNs) are benign tumours that develop in 20% to 50% patients, and will result in moderate-to-severe morbidities such as pain, motor dysfunction and disfigurement.
According to AstraZeneca, the potential benefit of selumetinib in NF1 is being examined in the Phase I/II SPRINT trial in paediatric patients with inoperable NF1-related PNs.
AstraZeneca global medicines development executive vice president and chief medical officer Sean Bohen 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.”
Selumetinib, which is an MEK 1/2 inhibitor and potential new medicine, is licensed by AstraZeneca from Array BioPharma in 2003.
In 2017, AstraZeneca entered into a co-development and co-commercialisation agreement for selumetinib and PARP inhibitor Lynparza for multiple cancer types.
The partnership will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies.
The NF1 gene offers instructions to make a protein known as neurofibromin that negatively controls the the RAS/MAPK pathway, enabling to control cell growth, differentiation and survival.
Mutations in the NF1 gene will cause dysregulations in RAS/RAF/MEK/ERK signaling, which will support cells to grow, divide and copy themselves in an uncontrolled manner and result in tumour growth.
Selumetinib suppresses the MEK enzyme in this pathway to help inhibit tumour growth. It is also being studied as a monotherapy and in combination with other treatments in other ongoing trials.
Merck Research Laboratories global clinical development head and senior vice president Roy Baynes 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 MSD and our partner AstraZeneca to help patients impacted by this debilitating genetic condition.”