(Alliance News) - AstraZeneca PLC on Tuesday said its drug selumetinib has been granted orphan drug designation in Japan as a treatment for the rare genetic disease neurofibromatosis type 1.
Cambridge-based pharmaceutical company Astra is co-developing and co-commercialising selumetinib with Merck & Co Inc.
Shares in Astra were down 0.8% at 8,477.00p in London in morning trading. Merck & Co's shares ended 1.2% higher in New York at USD76.12 on Monday and were up 0.7% in Tuesday's pre-market trading.
The drug treats neurofibromatosis type 1, symptoms of which include soft lumps on and, under the skin, skin pigmentation. In between 30% and 50% of patients, the disease also causes plexiform neurofibromas, where tumours develop on the nerve sheaths and can cause pain, visual impairment, disfigurement, airway dysfunction, and other serious symptoms.
The phase 1/2 Sprint Stratum 1 trial, sponsored by the National Cancer Institute's Cancer Therapy Evaluation Program, showed an overall response rate of 66% in paediatric patients with neurofibromatosis type 1 and plexiform neurofibromas when given selumetinib twice per day as an oral therapy.
The overall response rate was defined as the percentage of patients showing a confirmed or partial response of at least a 20% reduction in tumour volume.
The Japanese Ministry of Health, Labour & Welfare grants orphan status to medicines that are intended to treat diseases affecting less than 50,000 patients in Japan where there is a high unmet medical need.
Selumetinib has already been approved in the US under the brand name Koselugo to treat neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas on patients aged two years and older.
The marketing authorisation application for selumetinib in neurofibromatosis type 1 with plexiform neurofibromas has been accepted for review by the European Medicines Agency and further regulatory submissions are underway worldwide.
Jose Baselga, executive vice president of Oncology R&D at Astra, said: "Neurofibromatosis type 1 can have a devastating impact on children and new medicines are urgently needed to help treat the resulting plexiform neurofibromas and associated clinical issues. Current options 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."
By Anna Farley; annafarley@alliancenews.com
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