AstraZeneca and Merck (MSD) have secured the European Medicines Agency orphan designation for a MEK 1/2 inhibitor called selumetinib to treat neurofibromatosis type 1 (NF1).

NF1 is an incurable genetic disorder known to impact one in 3,000 newborns globally. The condition is characterised by mild-to-moderate symptoms affecting the skin, nerves and skeleton.

Around 20-50% of NF1 patients develop plexiform neurofibromas (PNs), which are benign tumours on nerve sheaths. The increase in these tumours causes moderate-to-severe morbidities, including pain, motor dysfunction and disfigurement.

Selumetinib’s inhibition of the MEK enzyme in the RAS/MAPK pathway helps to block tumour growth.

The drug was licensed by AstraZeneca in 2003 from Array BioPharma. Later in 2017, AstraZeneca signed an agreement with Merck to jointly develop and commercialise the product.

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.

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By GlobalData
“The granting of an orphan designation is a positive step forward for children with NF1.”

“The granting of an orphan designation is a positive step forward for children with NF1 and their families.”

Selumetinib is being investigated as a monotherapy and in combination with other treatments. Currently, the drug is in Phase I/II SPRINT trial in paediatrics suffering from inoperable NF1-related PNs.

MSD Research Laboratories said Global Clinical Development senior vice-president and head 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.”

Under the strategic oncology alliance, AstraZeneca and Merck plan to develop selumetinib as a treatment for various other types of cancer.

The partners will further assess the drug in conjunction with their respective PD-L1 and PD-1 candidates.