New Medicines

Type 1 neurofibromatosis (NF-1) in children


New molecular entity

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Pre-registration (Filed)
Dec 19 · EU filing for selumetinib has been made via the centralised procedure [12].
Dec 19 · EU filings planned for H1 2020. Selumetinib is listed in the pipeline with orphan and breakthrough designation in EU and US [10].
Nov 19 · Filed in the US with priority review status. Prescription Drug User Fee Act (PDUFA) date is set for the Q2 2020 [11].
Apr 19 · Granted Breakthrough Therapy Designation (BTD) in the US for the treatment of paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic or progressive, inoperable plexiform neurofibromas (PN) [6].
Dec 18 · Filings now expected H2 2019 [5].
Sep 18 · Has orphan drug status in EU & US [4].
Sep 18 · Filings planned for H1 2019 [2].


Selective and uncompetitive MEK kinase inhibitor
Type 1 is the more common form and caused by a defect in the gene, NF1, situated at chromosome 17q11.2. Both type 1 and type 2 NF are inherited as autosomal dominant conditions but, for both types, there is no family history in about 50%, reflecting the incidence of new mutations. The birth incidence of type 1 is given as 1 in 2,500-3,000 but may be slightly higher because of failure to diagnose milder cases [1].
Type 1 neurofibromatosis (NF-1) in children

Further information

To be confirmed

Trial or other data

Sep 19 · Great Ormond Street and AstraZeneca initiate PI/II (NCT03326388) open-label study to evaluate the early and late toxicities associated with intermittent dosing of selumetinib. The trial will test the effectiveness of the drug in reducing the size of plexiform neurofibromas and optic pathyway gliomas in children with NF1. It will also test the effectiveness of the drug in improving the participants function in day to day life [9].
Sep 19 · PI/II SPRINT study meets its primary endpoint [8].
Jan 19 · Data readout from the SPRINT study anticipated [7].
Dec 16 · Last patient commenced dosing in PI/II SPRINT study [5].
Sep 11 · PI/II SPRINT single-arm study will enrol 104 children with NF1 and inoperable plexiform neurofibromas (PN) (NCT01362803) in the US. Collection of primary outcome data (max. tolerated dose, tolerability & response rate) ies expected to complete Sep 20 [2,3].

Evidence based evaluations