AstraZeneca gains approval for Koselugo in rare neurological disorder after 17 years of development

Following the news that AstraZeneca’s Koselugo (selumetinib) has been approved by the US Food and Drug Administration (FDA) for the treatment of a rare neurological disorder;

Philippa Salter, Neurology & Ophthalmology Analyst at GlobalData, a leading data and analytics company, offers her view:

“Previous treatments for neurofibromatosis type 1 (NF1) in pediatric patients, who have symptomatic, inoperable plexiform neurofibromas, has been for managing symptoms only. As such, Koselugo has provided a major breakthrough in treatment for NF1 patients as it has demonstrated that it can shrink tumors and increase the quality of life for children with this disease. By 2026, GlobalData forecasts total annual revenue of $206m for Koselugo.

“Selumetinib was originally developed by Array BioPharma, and back in 2003 it announced a licensing and collaboration agreement to develop selumetinib with AstraZeneca. Since 2003, AstraZeneca has looked to develop selumetinib in several oncology indications, however, it has seen a string of clinical trial failures. A total 17 years later, the acquisition of selumetinib is finally paying out for AstraZeneca, which is now jointly developing selumetinib with Merck & Co, with the approval of Keselugo for its first indication.

“AstraZeneca will be looking for a fast rollout for Koselugo, which could be challenging given the current situation with COVID-19. However, as a rare disorder that had no treatment options, there are specific advocacy groups for NF1 that AstraZeneca can target. It is likely that patient awareness of the availability of this new drug will be high, which should help with the launch of Koselugo.”

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