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First Drug Approved for NF Patients in Europe

UPDATE JUNE 22, 2021
Koselugo (selumetinib) has been granted conditional approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in pediatric patients with neurofibromatosis type 1 (NF1) aged three years and above. It follows the recently announced positive recommendation (below) by the European Medicines Agency (Committee for Medicinal Products for Human Use), and last year’s landmark approval by the U.S. Food and Drug Administration.

This milestone moment in NF is the result of patients, families, researchers, clinicians, doctors, nurses, pharma, biotech, government, donors and friends and so many more working TOGETHER to #ENDNF. And we promise: we are not done. We will not stop until there are treatments for ALL types of NF! Click here to read the AstraZeneca announcement.


European Medicines Agency approves Koselugo to treat pediatric patients with NF1


APRIL 23, 2021 — The European Medicines Agency, the agency of the European Union in charge of the evaluation and supervision of medicinal products, has approved AstraZeneca’s Koselugo for the treatment of pediatric patients with neurofibromatosis type 1 (NF1) plexiform neurofibromas. The Agency’s Committee for Medicinal Products for Human Use (CHMP) made their announcement here.

This is a major milestone for patients living with neurofibromatosis (NF), a genetic disorder that causes tumors to grow on nerves throughout the body. Affecting 1 in 3,000 people of all populations equally, this announcement is the first ever approved treatment for NF in Europe, and portends the potential for the development of treatment options for all NF patients.

Koselugo, also known as selumetinib, is a MEK inhibitor that was first identified as a potential treatment for NF tumors in early-stage discoveries by Children’s Tumor Foundation (CTF)-funded researchers, who showed that MEK inhibitors could significantly affect NF tumor size. Positive early clinical results were first reported at CTF’s annual scientific NF Conference in 2015, as well as in subsequent publications in the New England Journal of Medicine in 2016 and 2020. Collaborative efforts among private and public partners led to a landmark clinical trial at the National Cancer Institute / National Institutes of Health (NCI/NIH), which showed that over 70% of participants exhibited tumor reduction from 20-60%. In April 2020, the U.S. Food and Drug Administration approved Koselugo for use in NF1 patients with inoperable plexiform neurofibromas. The approval and announcement from the European Medicines Agency will expand the availability of this promising treatment option to even more NF patients.

“We want to congratulate and thank our friends at AstraZeneca for this collaboration on behalf of NF patients, said Annette Bakker, PhD, President of the Children’s Tumor Foundation and Vice-Chair of Children’s Tumor Foundation Europe. “We are very hopeful that the approval of Koselugo by the European Medicines Agency will further motivate many pharma and biotech companies to join our movement of ending NF for the over 2.5 million patients who live with neurofibromatosis.”

“I am thrilled about the approval of Koselugo for European patients living with neurofibromatosis,” said Simone Manso, Chair of Children’s Tumor Foundation Europe. “The collaborative story leading to the availability of this treatment emphasizes that patient centric collaborations among various actors does pay off, with the fantastic outcome that kids with inoperable plexiform neurofibromas will benefit from a much-needed treatment. There is much we all still need to do for people living with NF, and we are convinced that the first available treatment will enable more to come.”

The Children’s Tumor Foundation (CTF) and Children’s Tumor Foundation Europe combined are the world’s largest nongovernmental funders of all forms of neurofibromatosis research in the world. Its collaborative and open-data research model has catalyzed new discoveries and in recent years helped triple the number of clinical trials for NF, identified new drug candidates for both NF1 and NF2, and established a unique worldwide consortium focused on pain in schwannomatosis. CTF is dedicated globally to finding treatments for the varying, devastating effects of NF, which can cause blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer.

To read AstraZeneca’s announcement, click here.