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(Alliance News) - The US Food & Drug Administration has accepted a new drug application and granted priority review for selumetinib as a potential new medicine for treatment of a rare genetic condition, neurofibromatosis type 1, AstraZeneca PLC and development partner Merck & Co Inc said Thursday.
Neurofibromatosis type 1 is a genetic condition that causes tumours to grow along nerves.
The pharmaceutical companies said the regulatory submission was based on positive results from Sprint Phase II Stratum 1 trial. An objective response rate was achieved in 66% of paediatric patients with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas when treated with selumetinib as a twice-daily oral monotherapy.
Selumetinib secured FDA breakthrough therapy designation in April, orphan drug designation in February 2018, EU orphan designation in August 2018, and Swissmedic orphan drug status in December 2018.
The drug would become the first medicine indicated for the treatment of paediatric patients with neurofibromatosis type 1 plexiform neurofibromas, if approved.
AstraZeneca shares in London were up 0.5% at 7,423.00 pence each on Thursday morning. Merck shares ended 0.8% higher on Wednesday at USD84.82 in New York.
By Tapan Panchal; tapanpanchal@alliancenews.com
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