AstraZeneca: Japan grants orphan drug status to selumetinib

(CercleFinance.com) - AstraZeneca said on Tuesday that Japanese regulators have granted orphan drug status to selumetinib to treat type 1 neurofibromatosis, a rare and debilitating genetic disease.


Japan grants orphan drug status to medicines intended for the treatment of diseases that affect fewer than 50,000 patients, and for which there is a high unmet medical need.

Patients with neurofibromatosis often experience tumours growing along their nerves, causing disfigurement, motor dysfunction, pain, visual impairment and bowel/bladder dysfunction.

Selumetinib is being jointly developed and commercialised with US peer Merck.

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