Merck: promising results from Koselugo study

(CercleFinance.com) - Alexion, AstraZeneca's rare disease company, and Merck announce positive results from the Phase 3 clinical trial of Koselugo in adult patients with neurofibromatosis type 1 (NF1) with symptomatic inoperable plexiform neurofibromas (PN).


Results showed that Koselugo significantly improved the objective response rate (ORR) compared to placebo, the primary endpoint of the study.

NF1 is a rare genetic condition affecting around 1.7 million people worldwide, 70% of whom are adults.

No treatment is currently approved for adults, leaving many patients to suffer debilitating symptoms.

The results indicate that KOSELUGO could reduce the size of plexiform neurofibromas and improve the management of adult patients, until now without an approved targeted treatment.

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