NICE recommends Koselugo for the treatment of neurofibromatosis type 1

The National Institute for Health and Care Excellence (NICE) has recommended AstraZeneca and MSD’s Koselugo (selumetinib) for use by the NHS in England as part of its marketing authorisation. This therapy is indicated for the treatment of symptomatic and inoperable plexiform neurofibromas (PN) associated with neurofibromatosis type 1 (NF1) in children 3 years of age and older.

NF1 is a debilitating genetic disease that affects one in 4,000 people worldwide. In 30-50% of people with NF, plexiform neurofibroma tumors grow on nerve sheaths and can lead to clinical problems, such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder dysfunction. or intestines.

Koselugo works by blocking specific enzymes – protein kinases mitogen-activated kinases 1 and 2, also known as MEK 1 and MEK2 – which are involved in stimulating cell growth. By blocking these enzymes, which are overactive in NF1, Koselugo slows the growth of tumor cells.

Vanessa Martin, President of the Childhood Tumor Trust, said: “Children with NF1 face many complications from their condition. Some may develop benign tumors called NP that can occur throughout the body, sometimes near vital organs and structures. These tumors can cause severe morbidity, including pain and disfigurement, motor dysfunction and, in some cases, life-threatening organ damage.

“Depending on their location, NPs can be extremely difficult or impossible to remove surgically and therefore are often left untreated. Each of these factors has a serious impact on the patient’s quality of life. Being able to medically treat NP has given hope and excitement to our community and marks an important step in the fight against the debilitating impact of these tumors.

“We understand the difficulties caused by the symptoms of NF1 and the implications this can have on a child’s overall well-being. We are therefore extremely pleased to see NICE welcoming selumetinib for the treatment of PN in children with NF1,” said Sean Richardson, CEO of Alexion. Director, UK and Ireland.

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