AstraZeneca PLC said its drug Koselugo won a key European regulatory backing for use in adults, a positive development for the company following recent setbacks in its drug pipeline.
The European Medicines Agency's advisory panel recommended the drug for the treatment of symptomatic, inoperable tumours in adult patients with neurofibromatosis type 1, a rare, progressive genetic condition.
The positive opinion was based on a late-stage trial where Koselugo showed a statistically significant objective response rate of 20% in tumour size reduction, compared to 5% with a placebo, the company said.
"The CHMP positive opinion of Koselugo in adults with NF1 PN builds on more than a decade of global clinical and real-world experience, reflecting the unmatched body of evidence supporting the safety profile and efficacy of Koselugo," said Marc Dunoyer, Chief Executive Officer of Alexion, AstraZeneca's rare disease unit.
The drug is already approved for use in children in the European Union and for adults in Japan and other countries, with further regulatory reviews ongoing, according to the company.
The news follows recent late-stage trial failures for the British drugmaker, including for an experimental rare-disease drug and a separate treatment for chronic obstructive pulmonary disease.
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