AstraZeneca Koselugo gets Chinese nod for paediatric patients with neurofibromatosis type 1, plexiform neurofibromas
In addition to China, Koselugo is also approved in the US, EU, Japan and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PNs.;
Cambridge: AstraZeneca has announced that Koselugo (selumetinib) has been approved in China for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.
The approval by the National Medical Products Administration (NMPA) in China was 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). The trial showed Koselugo, an oral treatment option, reduced the size of inoperable tumours in children.
NF1 is a rare, progressive genetic condition affecting one in 3,000 individuals worldwide, most commonly diagnosed in children under the age of 10. In 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas) and cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.
Li Qingfeng, Vice President of Shanghai Ninth People’s Hospital, JiaoTong University School of Medicine, Chairman of Department of Plastic and Reconstructive Surgery, said, “Children living with NF1 PN may face physical challenges and significant disruption to their daily lives, and early intervention is essential as tumour growth is often progressive and rapid, especially in the first decade of life. The approval of Koselugo in China has the potential to change the treatment trajectory of this debilitating genetic condition, providing a new option for addressing inoperable tumours by targeting the underlying cause of tumour growth through MEK inhibition.”
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