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Koselugo now available in Malaysia for paediatric patients with symptomatic neurofibromatosis type 1 (NF1) plexiform neurofibromas

KUALA LUMPUR, Malaysia, Dec. 6, 2023 /PRNewswire/ -- AstraZeneca's Koselugo (selumetinib) is now available in Malaysia as a monotherapy for the treatment of paediatric patients three years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN)1. Koselugo, which received regulatory approval earlier this year, is a product approved by the National Pharmaceutical Regulatory Agency (NPRA) for NF1 with inoperable PN.

NF1 is a rare and debilitating genetic condition.2. Some patients may develop PN which are tumours growing inside their nerve sheaths. These tumours can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bowel/bladder dysfunction and disfigurement.2,3,4

Vinod Narayanan, Country President, AstraZeneca Malaysia said: "We are excited that Koselugo is now available in Malaysia as it changes the lives of individuals suffering from NF1. NF1 is a genetically driven condition with an early onset that affects children. Those living with the condition may experience debilitating symptoms that impact their daily lives."

"By improving access to this innovative medicine, we can reduce the high unmet medical need of NF1 patients who have been struggling with a lack of treatment options. Ultimately, our mission is to transform the lives of people affected by rare diseases in Malaysia through the development and delivery of innovative medicines," Vinod concluded.

Results of a pivotal SPRINT trial showed a cPR of 70% (52 of 74 patients, confirmed partial response), where cPR is defined as a PR on consecutive restaging exams at least 3 months apart.5

KKLIU: 3147 

Tarikh Tamat Tempoh: 9 May 2024



NF1 is a debilitating genetic condition that affects individuals. 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)and tumours that develop on the nerve sheaths (plexiform neurofibromas). These plexiform neurofibromas can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder/bowel dysfunction.

PN begin during early childhood, with varying degrees of severity.


Koselugo (selumetinib) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).


AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit


1. Malaysian Koselugo Prescribing Information Version 3.0 April 2023.

2. Gross, Andrea M et al. "Selumetinib in Children with Inoperable Plexiform Neurofibromas." The New England journal of medicine vol. 382,15 (2020): 1430-1442. doi:10.1056/NEJMoa1912735

3. National Institute of Neurological Disorders and Stroke. Neurofibromatosis Fact Sheet. "What is NF1?". Available at: #3162_2 Accessed February 2020.

4. 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.

5. Gross, Andrea M et al. "Long-term safety and efficacy of selumetinib in children with neurofibromatosis type 1 on a phase 1/2 trial for inoperable plexiform neurofibromas." Neuro-oncology vol. 25,10 (2023): 1883-1894. doi:10.1093/neuonc/noad086 


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SOURCE AstraZeneca

By: PR Newswire Association LLC. - 06 Dec 2023
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