Recently the FDA approved AstraZeneca’s KOSELUGO™ (selumetinib) for treatment of pediatric patients two years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).
In support of the application, AstraZeneca’s submitted a natural history study of NF1, Study 01-C-0222 (NCT00021541; n=62). According to an FDA spokesperson, the results of the natural history study were supportive because the study “…provided sufficient evidence that spontaneous tumor regression or spontaneous reductions in PN-related morbidities do not occur as part of the natural history of disease, the observed effects (on NF1-related PN shrinkage) in the SPRINT trial could be reliably attributed to the selumetinib treatment effect.” However, the FDA did not find the natural history study to be suitable for any comparative analyses. Read more here.
(Source: Christina Purpura and Elizabeth Dabrowski; Aetion; 10/16/20)