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AstraZeneca has announced that a recent study has shown its brand of selumetinib (Koselugo) is effective in treatment of adult patients suffering from neurofibromatosis.

Selumetinib is a kinase inhibitor that inhibits the enzymes MEK1 and MEK2 that are involved in stimulation of cell growth.

In neurofibromatosis type 1 (NF1), these enzymes are overactive, causing tumor cells to grow in an unregulated way leading to development of plexiform neurofibromas.

Therefore, selumetinib slows down the growth of tumor cells and plexiform neurofibromas through MEK1 and MEK2 blockade.

With no approved treatment options for NF1, this could spell good news for these patients who suffer disfigurement, dysfunction, persistent pain, or endure multiple surgeries.

“With limited options to manage NF1 PN in adults, many patients experience functional impairment and symptoms, which can substantially impact their lives. These clinically meaningful data show Koselugo has the potential to make a positive impact in patient care by reducing the size of plexiform neurofibromas,” emphasized Prof. Ignacio Blanco Guillermo, MD, PhD, Chairman of the Spanish National Reference Centre for Adult Patients with Neurofibromatosis.

The phase III study coded KOMET found that in adults with neurofibromatosis type 1 who presented with inoperable, symptomatic plexiform neurofibromas (PNs), selumetinib either led to disappearance of the PNs or at least reduced the tumor volume by 20%.

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Thereby demonstrating a statistically significant and clinically meaningful objective response rate compared to placebo.

“These promising results demonstrate that Koselugo, the first and only approved targeted therapy for certain children with NF1 PN, now has the potential to benefit adult patients for whom there are no approved targeted therapies. As the largest and only global placebo-controlled Phase III trial in adults with NF1 PN, KOMET reinforces our leadership in advancing potential treatment options for people living with this debilitating disease. We look forward to sharing these findings with regulatory authorities,” stated Marc Dunoyer, Chief Executive Officer, Alexion, AstraZeneca Rare Disease.

The trial was conducted in 13 countries across Europe, Asia, South America, North America, and Australia, recruiting 145 adult patients whose baseline characteristics represented the global patient population with neurofibromatosis type 1.

In the US, EU, Japan, and other countries, Koselugo—developed by AstraZeneca and MSD in a collaboration agreement—has been approved for the treatment of certain pediatric patients with NF1 who have symptomatic, inoperable PN.

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