- AstraZeneca and MSD said their neurofibromatosis type 1 treatment had been granted breakthrough therapy designation from the US Food and Drug Administration. Mutations in the neurofibromatosis type 1, or NF1, gene may result in dysregulations in RAS/RAF/MEK/ERK signalling, which can cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumour growth.

Selumetinib, a potential treatment for NF1, inhibited the MEK enzyme in RAS/MAPK pathway, helping to control cell growth, differentiation and survival, potentially leading to inhibition of tumour growth.

Selumetinib was being assessed as a monotherapy and in combination with other treatments in ongoing trials, the company said.

Story provided by