New! SYMDEKO and KALYDECO information

We are thrilled to hear that Symdeko and Kalydeco for 12-24 months has been submitted to the PBAC for the next agenda with an outcome expected mid August. Now is the time to join together and make our voices and heard to ensure that people with CF stop falling through the cracks. Below we have listed key facts that you can refer to when talking about Orkambi, sample letters for you to send to your local, state and federal politicians, links to the PBAC portal for consumer comment and important dates surrounding this submission. 

• Key facts
• PBAC Portal key points
• PBAC links
• Sample Letter to Ministers
• Important dates

Key facts:

KALYDECO for children 12 to 24 months

• KALYDECO (ivacaftor) is safe and effective in children between the ages of 1 and 2 year with cystic fibrosis (CF) and is a prescription medicine for patients who have at least one gating mutation in their CF gene.
• KALYDECO improved sweat chloride levels; pancreatic function and was well tolerated by the trial cohort.
• Vertex’s PBAC submission if approved will allow young Australians with a gateing mutations to access KALYDECO. In Australia, KALYDECO is already approved for children with CF ages 2 and older who have one of 10 specific mutations.
• KALYDECO is known as a ‘potentiator’ and helps open the CFTR chloride channel. It is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
• Data from Phase 3 clinical trials in infants and toddlers reported that safety was similar to those seen in studies of older children.
• The effectiveness of the treatment was indicated by the improvement in sweat chloride levels
• The Phase 3 trial ARRIVAL was an open label study for children under the age of two with one of 10 ‘gating mutations’ (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). It was performed in two parts
  • In the first part of the study, children are treated with KALYDECO for five days to allow researchers to assess the treatment’s safety.
  • Patients who complete the first part could then continue in the second part, which involves treatment for 24 weeks.
• ARRIVAL recruited patients from the U.S., Australia, Canada, Ireland and the U.K. and not only looked at safety and pharmacokinetics (how the drug behaves in the body) over the six months of treatment but also researchers studied KALYDECO’s effectiveness.
• Pancreatic insufficiency is also the most common gastrointestinal complication of CF and KALYDECO improved pancreatic function and was generally well-tolerated with a safety profile similar to that seen in studies children 2+ years.
• The majority of adverse events were mild or moderate in severity and no patients discontinued KALYDECO during the trial due to adverse events. The most common adverse events were cough, fever, rash, elevated liver enzymes and rhinorrhea (runny nose).
• Serious adverse events were reported in two patients. One patient had cough that was treated with IV antibiotics and one patient had a viral infection followed by distal intestinal obstruction syndrome and constipation.
• KALYDECO for children with CF between the ages of 12 and 24 months has already been approved in US and Europe.
• “Cystic fibrosis is a progressive disease with organ damage already present at birth, so the earlier patients can begin treatment, the greater the potential for improved outcomes,” Nettie Burke CEO of Cystic Fibrosis Australia said today. “The ARRIVAL trial results are an important step in our goal of treating children as early as possible to slow down and modify the progression of CF” she added.

SYMDEKO for people with CF 12+

• SYMDEKO (tezacaftor/ivacaftor) for people with cystic fibrosis (CF) aged 12 and older has been submitted to the PBAC for reimbursement by Vertex and will be evaluated at the March 2019 meeting.
• SYMDEKO is a new treatment option for patients with the most common mutation in cystic fibrosis (two copies of the F508del mutation) and one copy of one of 14 mutations that result in residual CFTR activity (P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T)
• In addition, Vertex has submitted Symdeko to the Life Saving Drugs Program due the rarity of the mutations it can treat. This orphan designation for SYMDEKO will benefit those with one F508del mutation and one residual function mutation (F508del/RF).
• Orphan designation is only granted to either address an existing unmet need or can provide significant benefit for people with life-threatening or chronically debilitating diseases, affecting a small number of patients.
• SYMDEKO is also a great alternative for those who do not tolerate ORKAMBI and has proven to have less ‘drug on drug’ interactions than the alternative.
• The two randomized, double-blind, placebo-controlled trials in patients 12 years and older resulted in patient-reported improvements in outcomes beyond just improved respiratory symptoms.
• People with CF health perceptions and physical functioning improved.
• SYMDEKO was generally well tolerated and had less increased respiratory adverse events compared to placebo.
• The improvements in lung function were statistically significant and showed a mean absolute change in ppFEV₁ compared to placebo of 4.0 percentage points.
• The most common adverse reactions experienced by patients were headache, nausea, sinus congestion, dizziness and nasopharyngitis (the common cold).
• SYMDEKO is a combination of tezacaftor and ivacaftor. Tezacaftor is designed to address the movement and processing defect of the CFTR protein to enable it to reach the cell surface where ivacaftor (KALYDEKO) can increase the amount of time the protein stays open to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.
• SYMDEKO was approved by the FDA in the US in February 2017 and approved in Canada in June 2018.
• “SYMDEKO is a great breakthrough in CF care and will add at least 20 years to a person’s life let alone allow people with CF to be valuable and active members of our community” Nettie Burke CEO of Cystic Fibrosis Australia (CFA) said today. CFA will continue relentlessly to advocate for the best drugs and treatments for people with CF and in an attempt to leave no one behind we will always have a focus on the rare and difficult to treat mutations” she said.

PBAC Portal Key Points

1. We’re thrilled that Vertex has submitted two drug indications to the March PBAC meeting and we support the KALYDECO (1-2 year olds) and SYMDEKO (12+ years) listings.
2. CFTR Modulators like KALYDECO and SYMDEKO are designed to treat the underlying cause of CF. They are a first!
3. Until recently the treatment arsenal for cystic fibrosis was limited to therapies that treated the symptoms and clinical manifestations of the disease.
4. CFTR Modulators like KALYDECO and SYMDEKO are the first treatment available to patients that can prevent permanent, irreversible lung damage.
5. CF patients experience a 1-3% average decline in lung function per year. Maintaining or improving lung function has a significant clinical benefit.
6. Even when a CF patient does everything right, they will still experience recurrent pulmonary exacerbations.
7. People with CF have a fundamental medical need for modulating therapies like KALYDECO, ORKAMBI and SYMDEKO to correct the function of the CFTR protein.
8. For F508del mutation, patients need both a corrector (lumacaftor or tesacaftor) and a potentiator (ivacaftor).
9. FEV1 improvement varied greatly among patients in the clinical trials; some patients experienced dramatic improvement, some maintained their lung function, some improved modestly. All of these circumstances are clinically meaningful in CF.
10. CFTR Modulators can significant reduce pulmonary exacerbations.
11. Other positive findings included improvements in BMI and patient-reported outcomes.
12. The long term potential of KALYDECO and SYMDEKO are significant for both adults and children with CF. CFA will use the Australian Cystic Fibrosis Data Registry to gather information on efficacy and safety going forward.
13. Patient responses to CFTR Modulators will vary and can manifest in many different ways. Clinicians are best suited to determine whether the drugs are appropriate for each patient.
14. We urge the PBAC to evaluate the totality of evidence of patient response, including but not limited to improvements in FEV₁, reductions in pulmonary exacerbations, increases in BMI, reductions in hospitalizations, and improvements in mental health and patient-reported outcomes.
15. We must hold our politicians and bureaucrat’s consciences to the fire so they don’t shy away from the tough decisions.
16. We’re thrilled that Vertex has submitted two drug indications to the March PBAC meeting and we support the KALYDECO (1-2 year olds) and SYMDEKO (12+ years) listings.
17. CFTR Modulators like KALYDECO and SYMDEKO are designed to treat the underlying cause of CF. They are a first!
18. Until recently the treatment arsenal for cystic fibrosis was limited to therapies that treated the symptoms and clinical manifestations of the disease.
19. CFTR Modulators like KALYDECO and SYMDEKO are the first treatment available to patients that can prevent permanent, irreversible lung damage.
20. CF patients experience a 1-3% average decline in lung function per year. Maintaining or improving lung function has a significant clinical benefit.
21. Even when a CF patient does everything right, they will still experience recurrent pulmonary exacerbations.
22. People with CF have a fundamental medical need for modulating therapies like KALYDECO, ORKAMBI and SYMDEKO to correct the function of the CFTR protein.
23. For F508del mutation, patients need both a corrector (lumacaftor or tesacaftor) and a potentiator (ivacaftor).
24. FEV1 improvement varied greatly among patients in the clinical trials; some patients experienced dramatic improvement, some maintained their lung function, some improved modestly. All of these circumstances are clinically meaningful in CF.
25. CFTR Modulators can significant reduce pulmonary exacerbations.
26. Other positive findings included improvements in BMI and patient-reported outcomes.
27. The long term potential of KALYDECO and SYMDEKO are significant for both adults and children with CF. CFA will use the Australian Cystic Fibrosis Data Registry to gather information on efficacy and safety going forward.
28. Patient responses to CFTR Modulators will vary and can manifest in many different ways. Clinicians are best suited to determine whether the drugs are appropriate for each patient.
29. We urge the PBAC to evaluate the totality of evidence of patient response, including but not limited to improvements in FEV₁, reductions in pulmonary exacerbations, increases in BMI, reductions in hospitalizations, and improvements in mental health and patient-reported outcomes.
30. We must hold our politicians and bureaucrat’s consciences to the fire so they don’t shy away from the tough decisions.

PBAC Links:

• View the Agenda
• Submit your comment to the Consumer Portal (closes: 5pm, Wednesday 13 February 2019)

Important dates:

• 19 December 2018 - Consumer Portal opens for comment. 
• 15 January 2019 - Consumer Connect session with Jo Watson, Consumer Representative, PBAC. Learn more>
• 13 February 2019 – Consumer Portal Closes for comment.
• 13-15 March 2019 – PBAC Meeting in Canberra.
• 26 April 2019 – PBAC Announcement.

About CFA's Advocay plan and strategy

Cystic Fibrosis Australia has an advocacy strategy designed to ensure …

• cystic fibrosis is kept top of mind with our politicians and decision makers
• researchers are professionally drawn to the CF field and become involved
• funds are forthcoming from the corporate and community sectors for a worthwhile cause and
• the broad Australian community understands the inequity that surrounds CF.

CFA is a robust and authoritative voice for people with cystic fibrosis and their families, carers, partners and support networks. We are often speaking for the powerless to the powerful.

Government, business and the health and community sectors need to understand and appreciate the challenges people with CF face. This includes key issues such as drug affordability and availability, infection control, gene testing and access to the best medical and allied health services.

Over the past 18 months CFA has driven the following advocacy campaigns.