(Alliance News) - AstraZeneca PLC on Tuesday said its drug Koselugo has been approved as a treatment for children in the European Union with neurofibromatosis type 1 and plexiform neurofibromas.
Koselugo treats neurofibromatosis type 1, symptoms of which include soft lumps on and under the skin, and 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 approval was granted by the European Commission for children aged three and above and was based on positive results from the SPRINT Stratum 1 Phase 2 trial.
The 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 reduced the size of inoperable tumours in children, reducing pain and improving quality of life.
Cambridge-based pharmaceutical company AstraZeneca has been co-developing and co-commercialising Koselugo, also known as selumetinib, with Merck & Co Inc.
Shares in AstraZeneca were down 1.5% at 8,282.00p in London in morning trading.
Koselugo has already been approved in the US to treat neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas on patients aged two years and older.
"As the first medicine approved in the EU for patients with neurofibromatosis type 1, Koselugo has the potential to transform the way plexiform neurofibromas are managed and treated," commented Dave Fredrickson, executive vice president of the oncology business unit.
"The SPRINT data showed that Koselugo not only shrank tumours in some children, but also reduced pain and improved their quality of life."
By Will Paige; willpaige@alliancenews.com
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