- Pharma giant AstraZeneca said its drug to treat neurofibromatosis type 1, a rare and debilitating genetic disease, had been granted orphan drug designation in Japan.

Selumetinib, neurofibromatosis type 1 (NF1), was co-developed and co-commercialised with Merck.

The Japanese Ministry of Health, Labour and Welfare grants orphan drug designation to medicines intended for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there was a high unmet medical need.

'Current options (to treat NF1) in most countries are limited and this designation is a significant step forward in bringing the first medicine for NF1 to paediatric patients in Japan,' said José Baselga, executive vice president, oncology R&D.

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