UK major AstraZeneca (AZ, NASDAQ: AZN) has announced that its mitogen-activated protein kinase (MEK) inhibitor, Koselugo (selumetinib), has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromatosis (PN) in pediatric patients with neurofibromatosis type 1 (NF1) aged three years and above. This marks a significant advancement in the therapeutic options available for children affected by this condition.
Koselugo: The First and Only Approved Therapy in China for Pediatric NF1-Related PN
Koselugo is the first and only approved therapy by China’s National Medical Products Administration (NMPA) for the treatment of symptomatic, inoperable PNs in pediatric patients with NF1. It works by blocking specific enzymes (MEK1 and MEK2) that are overactive in NF1, leading to unregulated tumor cell growth. By inhibiting these enzymes, Koselugo slows down the growth of tumor cells.
Global Regulatory Landscape and SPRINT Trial Results
Koselugo is approved for use in the US, EU, Japan, and now China, and has received Orphan Drug Designation in the US, EU, Russia, Switzerland, South Korea, Taiwan, Japan, and Australia. Health authorities worldwide are reviewing regulatory submissions. The approval in China is based on positive results from the SPRINT Stratum 1 trial, sponsored by the National Institutes of Health’s National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP), which showed that the drug reduced the size of inoperable tumors in children.
AstraZeneca’s Strategic Collaboration with Merck for Oncology
In July 2017, AstraZeneca and US firm Merck & Co., Inc., announced a global strategic collaboration to co-develop and co-commercialize Lynparza and Koselugo for multiple cancer types. This partnership underscores the commitment to expanding the reach and impact of these therapies in the oncology field.-Fineline Info & Tech
