PBAC Approves ORKAMBI!

WA families rejoice the approval of a lifesaving drug

Melissa Gibson is cheering very loudly. Today Melissa and husband Justin are celebrating the Pharmaceutical Benefits Advisory Committee (PBAC) much awaited announcement that a deal with Vertex Pharmaceuticals for access to a life-saving drug called Orkambi® for son Toby has finally been achieved.

For Toby, who turned eight in July, the announcement is hard to comprehend. Toby has cystic fibrosis (CF), a rare genetic condition predominantly affecting the lungs and pancreas. A life shortening condition, with a relentless daily regime, there is currently no cure for CF.

Toby was born with two copies of the F508del gene mutation that Orkambi® targets. Mum, Melissa, says “The availability of Orkambi® will be life altering for Toby, as it will allow him to experience a better quality of life through improved lung function and less hospital admissions. Orkambi will also help prevent further lung damage.”

Nigel Barker CEO of Cystic Fibrosis Western Australia (CFWA), says “The decision is a culmination of years of advocacy by people whose lives have been impacted by CF to force an agreement between the government and Vertex, the manufacturer of Orkambi.®”

Today’s announcement means children 6 and over and adults living with CF who have two copies of the F508del gene mutation will finally have access under the PBS pricing.

Nigel adds “We are ecstatic that the Australian Health System is supporting better health outcomes and quality of life for those with rare diseases, but we’re also concerned that lives have been put on hold and lungs have incurred irreversible damage whilst we struggle to get a fair deal, years behind approval in the USA and Europe. There has to be a better way to fast track the process of drug approval in the future.”

CF affects nearly 4,000 Australian children and adults; today’s announcement will be life changing for those that will now have access to Orkambi®.

For CFWA President, Caz Boyd, who was born with two copies of the F508del gene mutation that Orkambi® targets and underwent a lung transplant at the age of 27, the announcement is exciting. “I am grateful that for a large group of youngsters living with CF their journey will be different to mine; if this drug was available 24 years ago I wouldn’t have needed a lung transplant and witnessed the lives of many childhood CF friends cut short by this insidious disease.  We were confident that the PBAC and Federal Government understood the needs of people with CF. Today’s announcement continues to reflect that Australia’s health system remains one of the best in the world.”

The CF community applauds the outcome of today’s decision, whilst recognising that this is part of a range of outcomes that are needed to improve the lives of people living with CF.

Tomorrow CFWA and families impacted by CF, along with CFWA researchers and funding partners will continue their journey to improving the lives of children and adults living with CF with the aim of achieving a vision of Lives unaffected by CF.

If you would like to speak to one of our CFWA team about the impact of this announcement on you or your family member please contact our office at servicesmanager@cfwa.org.au or on 6457 7333.

For more information please visit the PBS website.

More Information

  • Cystic fibrosis (CF) is a genetic disease, which affects nearly 4,000 children and young adults living in Australia.
  • It is as common as childhood leukaemia but whereas over 95% of cases of childhood Leukaemia are cured, there is still no cure for Cystic Fibrosis.
  • Over one million adults in Australia carry the recessive gene and don’t know it.
  • A baby born to two carriers has a 1 in 4 chance that he or she will be born double recessive and have Cystic Fibrosis.
  • Mutations in the cystic fibrosis gene interfere with the manufacture of a protein responsible for regulating the water content of cells.
  • There is no cure for cystic fibrosis.
  • Orkambi® is a prescription medicine used for the treatment of CF in patients who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. Orkambi® is a new generation of CF drugs that treats the disease not the symptoms.
  • Clinical trials show that
    • Lung function was preserved, and further decline was halted, relative FEV1 increased by 4.8% and a sustained improvement was evident
    • Exacerbations reduced, as did hospitalisations and antibiotic use.
      • Reduced Exacerbations by 39%
      • Hospitalisations by 61%
      • Antibiotic use reduced by 56%
    • Nutrition improved, as did BMI and diabetes is less likely or less severe symptoms will be experienced.
    • Mental health and quality of life improved. Social inclusion, employment and education participation increase. Personal and family stress was reduced resulting in less depression and anxiety for patients, parents and support networks
  • The Orkambi® approval will:
    • provide relief for around 1,000 Australians who have two copies of the F508del gene mutation.
    • together with Kalydeco® will mean over half of the CF population will have access to medication that can change the course of their condition.
    • However, many families still face agonising challenges. Orkambi® and previously approved drug Kalydeco® now account for just over half of CF mutations, however these drugs do not work for everyone affected.

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