AstraZeneca Plc (LON:AZN), a global, science-led biopharmaceutical company, and global pharmaceutical company Merck & Co Inc (NYSE:MRK) (known as MSD outside the US and Canada) announced on Friday the grant by the European Medicines Agency (EMA) of orphan designation to selumetinib, a MEK 1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1).
NF1 is an incurable genetic condition that affects one in 3,000 newborns worldwide. Currently, there is no cure for NF1, a life-long and devastating condition and current treatment choices for these patients are very limited.
Reportedly, the potential benefit of selumetinib in NF1 is being explored in the phase I/II SPRINT trial in paediatric patients with inoperable NF1-related PNs. Select findings were presented recently at the 2018 American Society of Clinical Oncology Annual Meeting in Chicago by the principal investigators at the National Cancer Institute. Full results are expected later in 2018.
Orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan status by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is seriously debilitating/life threatening and has a prevalence of up to five in 10,000 in the European Union.
Also, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan designation is conferred following a positive opinion by the EMA's Committee for Orphan Medicinal Products.
Selumetinib was granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of NF1 in February 2018.
In July 2017, AstraZeneca and Merck & Co Inc US (MSD outside the US and Canada)had announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world's first PARP inhibitor and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types.
Under this collaboration, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as monotherapies. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.
AstraZeneca focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in the three therapy areas of Oncology, Cardiovascular, Renal & Metabolism and Respiratory.
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