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Alyftrek (vanzacaftor, tezacaftor, deutivacaftor)
Australian Prescription Medicine Decision Summary
Alyftrek is a new prescription medicine for people aged six and above who have cystic fibrosis. It combines 3 active ingredients that work together to improve breathing and overall health. Alyftrek contains the active ingredients vanzacaftor, tezacaftor, and deutivacaftor.
Published
Product name
Alyftrek
Active ingredient
Vanzacaftor, tezacaftor, deutivacaftor
Submission type
New chemical entity - Type A/B application
Decision
Approved for registration in the Australian Register of Therapeutic Goods (ARTG)
Decision date
Registration date
What this medicine was approved for
Alyftrek is a new prescription medicine to help people with cystic fibrosis who are 6 years and older. It has 3 active ingredients that target the genetic cause of cystic fibrosis.
This medicine is for people who have changes (mutations) in a gene called the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Changes in the CFTR gene can cause faulty production of the CFTR protein, or its functioning. CFTR protein helps maintain the balance of salt and water on many surfaces in the body, including the lungs.
In people with cystic fibrosis, CFTR protein defects result in the build-up of thick, sticky mucus that can make breathing difficult and permanently damage the lungs, digestive system and other organs.
Alyftrek is the first once-daily CFTR modulator and is indicated for a broad number of mutations in the CFTR gene. The active ingredients in Alyftrek (vanzacaftor, tezacaftor, deutivacaftor) work together to control the production of mucus. This helps the lungs and digestive tract to work better, keeping people with cystic fibrosis healthier.
How this medicine works
Alyftrek belongs to a group of medicines called CFTR modulators. Each tablet contains vanzacaftor, tezacaftor, deutivacaftor. These active ingredients improve CFTR protein quantity and function, helping chloride move in and out of cells as it should. Doctors can measure this improvement by testing how much chloride is in your sweat or by using other medical tests.
Studies show that Alyftrek helps to thin the mucus produced in the lung, improving lung function and breathing. Alyftrek may reduce how often a person with cystic fibrosis gets sick and help them maintain a healthy weight.
Why the TGA approved or did not approve this medicine
The main evidence of effectiveness and safety comes from 2 important clinical studies. These studies took place in many different hospitals and clinics around the world.
In both studies, researchers compared the effectiveness and safety of Alyftrek to Trikafta (elexacaftor, tezacaftor, ivacaftor, and ivacaftor), which is already approved for use in cystic fibrosis. The studies showed that Alyftrek maintained participants’ quality-of-life and number of CF-related lung exacerbations and hospital visits, similar to Trikafta.
Key findings from clinical studies
- Alyftrek was as effective as Trikafta at maintaining lung function in people with cystic fibrosis.
- On average, Alyftrek lowered the levels of chloride found in sweat more than Trikafta, no matter the patient’s genetic type of cystic fibrosis.
- The medicine worked well for people of all ages, lung health levels, and genetic types of cystic fibrosis.
- Alyftrek was generally safe and well tolerated in patients aged 6 years and older. Common side effects include headache and diarrhoea.
Other treatment-related side effects included raised liver enzymes, rash and raised blood levels of creatine kinase (an enzyme in the blood, which can indicate muscle damage). These events were manageable, causing low rates of treatment interruptions and discontinuations. For comprehensive information on the potential side effects and risks of Alyftrek, refer to the Consumer Medicine Information (CMI) leaflet or Product Information (PI) document (found via the below hyperlinks).
The TGA decided that the application has provided sufficient evidence supporting the safety and effectiveness of Alyftrek and the medicine can be registered for use in Australia.
More detailed information on why the TGA approved Alyftrek will be published in the upcoming Australian Public Assessment Report (AusPAR).
The information in this decision summary will not be updated to include any subsequent changes, so may not represent the most current information for a medicine. For current information, refer to the medicine’s CMI or PI.
Consumer Medicine Information (CMI)
The CMI leaflet offers guidance for consumers to support safe and effective use of the medicine. The CMI includes information on dose, how to use the medicine properly, potential side effects, safety precautions, storage instructions and more.
The Australian CMIs for Alyftrek can be accessed through the ARTG hyperlinks provided below.
Product Information (PI)
The PI document provides essential prescribing information for health professionals, including details on dosage recommendations, pregnancy category, contraindications, precautions and potential side effects.
The Australian PIs for Alyftrek can be accessed through the ARTG hyperlinks provided below.
Other resources
For health advice and information, including a symptom checker and service finder refer to the healthdirect website.
For advice on prescription medicines, over the counter medicines and other medicines (including complementary medicines) call Medicines Line.
For information on medicines subsidised by the Australian Government refer to the Pharmaceutical Benefits Scheme (PBS) website.
For data and reports on health and welfare topics in Australia refer to the Australian Institute of Health and Welfare website.