AstraZeneca PLC - Selumetinib granted Japan orphan drug designation
30 June 2020
Selumetinib granted orphan drug designation
Designation follows recent US approval
with additional regulatory submissions underway
Phase II SPRINT trial showed selumetinib reduced tumour
volume in paediatric patients with NF1 plexiform neurofibromas
AstraZeneca today announced that selumetinib has been granted orphan drug designation (ODD) in
Selumetinib is co-developed and co-commercialised with
Some 30-50% of patients with NF1 experience plexiform neurofibromas (PN) - tumours growing along their nerve sheaths.2 These PN can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bowel/bladder dysfunction.3
AstraZeneca and MSD are jointly developing and commercialising selumetinib which was approved in the US in
NF1 is a debilitating genetic disease that affects one in every 3,000 to 4,000 individuals.1 It is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called 'café au lait' spots)1 and, in 30-50% of patients, tumours develop on the nerve sheaths (plexiform neurofibromas).2 These plexiform neurofibromas can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.3 PN begin during early childhood, with varying degrees of severity, and can reduce life expectancy by 8 to 15 years.1,4,5
The SPRINT Stratum 1 Phase I/II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy.6 Results were published in
Selumetinib (available in the US under the medicine name Koselugo) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).6 MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.
Selumetinib was granted
AstraZeneca and MSD Strategic Oncology Collaboration
AstraZeneca in Oncology
AstraZeneca has a deep-rooted heritage in oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients' lives and the Company's future. With six new medicines launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca's main capabilities, the Company is actively pursuing innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in
By harnessing the power of four scientific platforms - Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates - and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.
AstraZeneca (LSE/STO/NYSE: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in
2. Hirbe AC, Gutmann DH. Neurofibromatosis type 1: a multidisciplinary approach to care. The Lancet Neurology. 2014;13:834-43. DOI: 10.1016/S1474-4422(14)70063-8.
3. Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med. 2016;375:2550-2560. DOI: 10.1056/NEJMoa1605943.
5. Evans DGR, O'Hara C, Wilding A, et al. Mortality in neurofibromatosis 1: in
6. Koselugo (selumetinib) [prescribing information].
7. Gross A, Wolters P, Dombi E, et al. Selumetinib in Children with Inoperable Plexiform Neurofibromas. N Engl J Med. 2020;382: DOI: 10.1056/NEJMoa11912735.
This information is provided by RNS, the news service of the
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