PBS expands access with new listings for rare diseases and cancers

Pharma News: Australians with rare diseases and rare cancers now have access to new medicines under the Pharmaceutical Benefits Scheme (PBS). These latest additions follow recent PBS listings of therapies from Bayer, BMS, Ipsen, Roche and Vertex.

Alexion’s Koselugo (selumetinib) is the first and only therapy now reimbursed to treat symptomatic, inoperable benign nerve tumours in children two years old and over with neurofibromatosis type 1 (NF1).

NF1 is a rare genetic disorder affecting about one in 3,000 Australians. Around 170 children are expected to benefit from this listing each year.

“Historically, the options to treat plexiform neurofibromas have been limited. Due to the unpredictability of PNs, standard management often involves regular monitoring for disease progression and complications, and pain management. Surgical options are complicated by the vascular and infiltrative nature of these tumours, and resection is rarely complete,” said Clinical Associate Professor Mimi Berman, President, Human Genetics Society of Australasia.

“For some children however, PN tumours can grow large enough to impact nearby tissues and organs, limiting their functioning, and causing debilitating pain and disfigurement. This impacts their quality of life including their mental health, education and social relationships. Combined with the ongoing burden of surgeries, as tumours continue to grow and regrow, the impact of PNs on children and their families is profound. We have urgently needed treatment options that target tumour growth,” concluded A/Prof Berman.

Leanne Dib, CEO, Children’s Tumour Foundation of Australia said “We are absolutely thrilled to see such important progress being made in Australia to ensure children with NF1 who have inoperable plexiform neurofibromas have access to this government-funded medication. These tumours can be painful, disfiguring and lead to malignancy, but the emotional, social and financial toll can be equally as burdensome. Not just for the child, but the whole family. I 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.”

Nicole Gaupset, General Manager, Alexion AstraZeneca Rare Disease, Australasia, said “At Alexion, our mission is to transform the lives of people impacted by rare diseases and we are committed to improving equitable access to innovative medicines.”

In related PBS news, Alnylam Australia’s Onpattro (patisiran) has been reimbursed for the first time to treat hereditary transthyretin-mediated (ATTR) amyloidosis that has affected many parts of the nervous system.

Hereditary ATTR amyloidosis is a rare and rapidly progressive disease that prevents the nerves, heart and other parts of the body from working normally. If left untreated, the disease can progress to potentially life-threatening complications and death within a few years after symptom onset.

This listing is expected to benefit around 55 patients each year.

Veriton Pharma’s Zyamis (midazolam) will be listed to provide Australians living with epilepsy with a solution delivered via pre-filled syringes for the emergency treatment of potentially life-threatening seizures.

Zyamis will be listed on the PBS to treat a form of epilepsy known as Generalised Convulsive Status Epilepticus (SE). SE is a prolonged or continuous seizure lasting more than 5 minutes, or a cluster of repeated seizures without full recovery in-between, and can be life-threatening or lead to long term consequences, such as permanent brain damage if not treated quickly.

More than 10,000 patients each year are expected to benefit from the PBS listing.

Mark Butler MP, Federal Minister for Health and Aged Care, said “These PBS listings are yet another example – providing new help and new hope to Australian kids and adults.

“The PBS, introduced by a Labor Government, sits alongside our other proud social reforms in the health space, notably, of course, Medibank and then Medicare, 40 years old this year.”

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