KOSELUGO™ (selumetinib) Approved for the Treatment of Neurofibromatosis Type 1-associated Plexiform Neurofibromas in Pediatric Patients, Available from Onco360
Onco360®, the nation’s largest independent Oncology Pharmacy, has been selected by AstraZeneca to be a specialty pharmacy partner for KOSELUGO™ (selumetinib), a novel oral kinase inhibitor indicated for the treatment of pediatric patients aged two years and above, with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas.
“The approval of KOSELUGO represents a new treatment strategy for children suffering from the debilitating effects of plexiform neurofibromas arising from NF1,” said Paul Jardina, President and CEO, Onco360. “As a specialty pharmacy dedicated to serving patients with cancer and other orphan and rare diseases, we are committed to improving the lives of pediatric patients suffering from neurofibromatosis type 1-associated plexiform neurofibromas.”
NF1 is a rare, progressive genetic disorder, occurring in approximately one in every 3000 live births, which can affect multiple organ systems and is characterized by changes in skin pigmentation, neurologic impairment, skeletal impairment, development of benign tumors, and development of malignant tumors. Unfortunately, 30-50% of NF1 patients develop plexiform neurofibromas, which are benign tumors of the nerves that form on or underneath the skin. Although plexiform neurofibromas may appear anywhere in the body, some of the most common locations of tumor involvement include the head, neck, trunk, extremities, and areas around the spine. Plexiform neurofibromas can cause the following symptoms dependent upon location: disfigurement, pain, motor dysfunction, visual loss, airway compromise, weakness, bowel dysfunction, bladder dysfunction, and mobility issues. Rarely, plexiform neurofibromas can transform into cancerous tumors such as sarcomas. The primary treatment for plexiform neurofibromas involves surgical resection. Unfortunately, many patients are not surgical candidates due to the location or volume of these tumors. Additionally, plexiform neurofibromas commonly recur following optimal surgical resection, thus representing a significant area of unmet medical need1.
KOSELUGO is manufactured by AstraZeneca, a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines, and was approved by the U.S. Food and Drug Administration (FDA) on April 10, 2020, to treat pediatric patients (aged two years and older) with NF1 and symptomatic, inoperable plexiform neurofibromas. The FDA’s approval of KOSELUGO is based on the results of the Phase II SPRINT trial (NCT01362803), which demonstrated a 66% objective rate of response1. Prior to the approval of KOSELUGO, there were no medications that were specifically FDA-approved for the treatment of pediatric patients with NF1-associated plexiform neurofibromas. For full prescribing information, visit KOSELUGO.com.