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EMA Accepts Submission for Koselugo (Selumetinib) as Treatment for Neurofibromatosis


EMA Accepts Submission for Koselugo (Selumetinib) as Treatment for Neurofibromatosis

Koselugo (approved in the US) on its way to Become First Approved Drug for Neurofibromatosis Type 1 (NF1) in Europe.


On April 10, AstraZeneca and MSD (Merck) announced that the U.S. Food and Drug Administration (FDA) has approved Koselugo (Selumetinib) for use in patients with plexiform neurofibromas, a common manifestation in the disease neurofibromatosis type one (NF1). The FDA’s approval is a major milestone for Koselugo becoming the first ever approved treatment for NF patients in the US.

On April 29, AstraZeneca and MSD (Merck) announced that the regulatory submission of Koselugo was accepted by EMA. Anticipated approval in Europe has been set for the first quarter of 2021.

This success follows comprehensive clinical testing (Sprint trial) of the drug in patients at the National Cancer Institute (NCI), a division of the National Institutes of Health (NIH). In those clinical trials, over 70% of NF patients with inoperable plexiform neurofibromas saw tumor size reduction anywhere from 20-55% in size. In addition to both visible and actual tumor reduction, patients reported higher-quality physical function, improved mobility, and enhanced emotional and psychological status.

The first use of MEK inhibitors as a potential treatment for NF tumors came from an early-stage discovery by Children’s Tumor Foundation (CTF)-funded researchers, who showed that the repurposed oncology drug selumetinib (Koselugo) could affect NF tumor size. Positive early clinical results were first reported at CTF’s annual scientific NF Conference in 2015, as well as in a subsequent publication in the New England Journal of Medicine.

Collaborative efforts among the NCI, the NIH, the NFRP-CDMRP (Neurofibromatosis Research Program of the Congressionally Directed Medical Research Programs), NTAP (Neurofibromatosis Therapeutic Consortium), and CTF ensured that this ‘MEK journey’ proceeded efficiently and expeditiously through proactive and strategic coordination, which included patient input in the mix. A soon-to-be-published paper on this unique collaboration, and its potential application for other disease areas, will appear in EMBO Molecular Medicine.

“Today’s announcement from AstraZeneca and MSD/Merck is a gigantic step towards our ultimate dream – approved treatments improving patients the quality of life for all forms and symptoms of neurofibromatosis,” said Simone Manso and Annette Bakker, PhD, Chair and Vice chair of the Children’s Tumor Foundation Europe. “We are very excited for the patients in Europe that they hopefully may also benefit soon from Koselugo. If the EMA approves Koselugo (as early as 2021 Q1), this treatment will open the door to increased interest in NF globally, as a validation of the power of working together to find cures for patients who most need them.”

Founded in 2018, the Children’s Tumor Foundation Europe was founded by the Children’s Tumor Foundation in the USA, the world’s largest nongovernmental funder of all forms of neurofibromatosis research in the world. Both foundations collaborate to build global bridges, globalize NF research and promote collaboration with common endgoal of catalyzing new discoveries and translating those discoveries into better and safer treatments for all NF patients (NF1, NF2, Schwannomatosis).

Neurofibromatosis is a genetic disorder that affects 1 in 3,000 births of all populations equally, and causes tumors to grow on nerves throughout the body. Its devastating effects can cause blindness, deafness, bone abnormalities, disfigurement, learning disabilities, disabling pain, and cancer.

To read Astra Zeneca Q1 results click here.

To read more about the MEK story collaboration, click here.

To view videos of the positive impact this treatment has on NF patients, watch Philip’s story here and Jane’s story here. Read the stories of other NF patients currently receiving this
treatment here.