AstraZeneca gets 'breakthrough' status for selumetinib

(Sharecast News) - AstraZeneca announced on Monday that the US Food and Drug Administration (FDA) has granted 'Breakthrough Therapy Designation' for the MEK 1/2 inhibitor and potential new medicine selumetinib, which it is developing in partnership with Merck & Co.The FTSE 100 pharmaceuticals giant said the designation was for the treatment of paediatric patients aged three years and older with neurofibromatosis type 1 (NF1) symptomatic or progressive, inoperable plexiform neurofibromas (PN), which it described as a rare, incurable genetic condition.It said the designation was based on phase 2 data from the SPRINT trial, testing selumetinib as an oral monotherapy in paediatric patients aged three years or older with inoperable NF1-related PN.The results of the trial were presented by the National Cancer Institute at the 2018 American Society of Clinical Oncology Annual Meeting.It was the ninth Breakthrough Therapy Designation that AstraZeneca had received from the FDA since 2014.The firm said such a designation was designed to expedite the development and regulatory review of medicines that were intended to treat a serious condition, and that have shown "encouraging" early clinical results, which could demonstrate substantial improvement on a clinically-significant endpoint over available medicines.Selumetinib was granted 'Orphan Drug Designation' for the treatment of NF1 by the FDA in February last year, and the European Medicines Agency in August.Selumetinib is a MEK 1/2 inhibitor, and potential new medicine licensed by AstraZeneca from Array BioPharma in 2003.AstraZeneca and Merck & Co entered a co-development and co-commercialisation agreement for selumetinib in 2017.The company explained that the NF1 gene provided instructions for making a protein called neurofibromin, which negatively regulated the RAS/MAPK pathway, helping to control cell growth, differentiation and survival.Mutations in the NF1 gene could result in dysregulations in RAS/RAF/MEK/ERK signalling, which in turn could cause cells to grow, divide and copy themselves in an uncontrolled manner, and may result in tumour growth.Selumetinib inhibited the MEK enzyme in that pathway, potentially leading to inhibition of tumour growth.It was being assessed as a monotherapy, and in combination with other treatments in ongoing trials."Selumetinib shows promise in the treatment of NF1-related plexiform neurofibromas, a rare and debilitating disease with no approved medications to date," said AstraZeneca's executive vice-president of research and development in oncology, José Baselga."The Breakthrough Therapy Designation acknowledges the significant unmet need of these patients and the potential benefit of selumetinib in this setting."Roy Baynes, senior vice-president and head of global clinical development, and chief medical officer, at Merck's MSD Research Laboratories, said the new designation validated the ongoing development of selumetinib."As a result of this, selumetinib has the potential to receive expedited regulatory review and we hope to bring this medicine to patients as soon as possible," Baynes added.