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LONDON (Alliance News) - AstraZeneca PLC on Monday said its drug selumetinib has been granted a breakthrough therapy designation in the US for neurofibromatosis type 1.
Neurofibromatosis type 1 is an incurable genetic condition affecting between 1 in 3,000 and 1 in 4,000 people. The disease can cause tumours called plexiform neurofibromas to develop on the the protective layer around the nerves.
These tumours may result in pain, disfigurement, difficulty controlling movement, and breathing problems. They can also become cancerous.
The breakthrough therapy designation applies to the treatment of paediatric patients who are at least three years old that have neurofibromatosis type 1 and plexiform neurofibromas.
The designation - which is intended to speed up the development and approvals process for drugs with encouraging trial results and significantly improve upon existing treatment - was awarded based on data from Astra's phase two Sprint trial. The trial found a 72% response rate in paediatric patients.
Jose Baselga, executive vice president of oncology research & development at Astra, said: "Selumetinib shows promise in the treatment of [neurofibromatosis type 1]-related plexiform neurofibromas, a rare and debilitating disease with no approved medications to date. The breakthrough therapy designation acknowledges the significant unmet need of these patients and the potential benefit of selumetinib in this setting."
Shares in AstraZeneca were up 1.9% at 6,250.00 pence on Monday morning.
