AstraZeneca's Koselugo (selumetinib) now available in Malaysia as a monotherapy for the treatment of paediatric patients three years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Koselugo, which received regulatory approval earlier this year, is a product approved by the National Pharmaceutical Regulatory Agency (NPRA) for NF1 with inoperable PN. NF1 is a rare and debilitating genetic condition.

Some patients may develop PN which are tumours growing inside their nerve sheaths. These tumours can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement. Results of a pivotal SPRINT trial showed a cPR of 70% (52 of 74 patients, confirmed partial response), where cPR is defined as a PR on consecutive restaging exams at least 3 months apart.