Koeulg for adults – pro
This drug is only currently approved for people between the ages of 2-18 with NF1 and symptomatic plexiform neurofibromas for which surgery is not feasible or advisable. EU has approved it only for pediatric use as well. The approval was based on results from the SPRINT Stratum 1 Phase II trial, which was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy. In the trial, Koselugo had an objective response rate (ORR) of 66 percent. ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20 percent reduction in tumour volume).
It is in a clinical trials, MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas, NCT02407405. The denial was appropriate based on it being in a clinical trial. Dose and effectiveness need to be confirmed.
Koselugo (selumetinib) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2020.
Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med. 2016;375:2550-2560. DOI: 10.1056/NEJMoa1605943.
Gross A, Wolters P, Dombi E, et al. Selumetinib in Children with Inoperable Plexiform Neurofibromas. N Engl J Med. 2020;382: DOI: 10.1056/NEJMoa11912735.