eligible Australian children now have access to the first medication for inoperable plexiform neurofibromas.
The PBAC has recognised the challenges patients and families face with complex NF tumours and the significant unmet need in treatment options.
More than a third of individuals with neurofibromatosis type 1 (NF1), develop a type of tumour known as plexiform neurofibromas (PNs).
Depending on where they form, these tumours can grow to become large and can cause debilitating pain, impact body functions like eyesight, breathing, motor or bowel function and in an estimated 10 per cent of cases, they can become malignant. The emotional, social and financial toll on families can be equally as burdensome.
Plexiform neurofibromas are difficult to treat and can regrow when surgically removed.
This first and only medication, Koselugo® (selumetinib) will target tumour growth, by acting as a MEK inhibitor that blocks specific enzymes (MEK1 and MEK 2) which are involved in stimulating cells to grow.
Thanks to years of advocacy, patient comments and the support of Alexion Pharmaceuticals Australasia, eligible children aged 2 years+ will now have access to this medication will now be available on the government funded Pharmaceutical Benefits Scheme (PBS). It is estimated that this will directly impact ~250 Australian children.
We are thrilled to see such important progress being made in Australia and would like to extend a heartfelt thanks to our community for sharing their experiences and to the Government for listening and providing hope for a better tomorrow.
To learn more about Koselugo® (selumetinib), you can read the PBS Website Listing or review the Consumer Medicines Information. Alternatively, please contact us at support@ctf.org.au or by phone 02 9713 6111 with any questions you may have.