MHRA: Kaftrio and Kalydeco licensed for patients aged two to five years old!
Written by Marc Cotterill
Resilient
In the wake of recent developments, it's been a challenging period, to say the least. Parents across the UK have been filled with concern and uncertainty regarding the accessibility of CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator therapies for their children.
When the draft recommendation from NICE was announced on the 3rd of November 2023, explaining that it does not recommend CFTR modulators, the anger and anxiety amongst the cystic fibrosis community was palpable. Thousands of families took to local, mainstream and social media to make sure the collective voice of the community was heard, and it has been inspiring to witness.
Whenever I'm asked to describe the CF community, the term 'resilient' immediately comes to mind. This is a community that bands together like a family, and it's all the more remarkable considering we often cannot meet in person due to the risk of cross-infection, where one individual with CF could inadvertently worsen another's condition, and vice versa. But it never stops us, we just get on and fight.
The threat of just missing out on these life-changing drugs was a real concern for families with young children on the cusp of the eligible age for Kaftrio, or those eagerly awaiting a decision from the MHRA (Medicines and Healthcare products Regulatory Agency) in hopes that the age limit would be lowered to match the EMA's (European Medicines Agency) guidelines.
But thankfully today—the 15th of November 2023—our community finally receives positive news, and the timing couldn’t be better.
A New Chapter Begins!
A new chapter begins today as we celebrate a significant breakthrough: the MHRA has officially licensed the cystic fibrosis drugs Kaftrio and Kalydeco for use in children aged two to five years old.
Here are the facts:
Licence Extension: The MHRA has extended the licence of cystic fibrosis medicines Kaftrio (ivacaftor, tezacaftor, elexacaftor) and Kalydeco (ivacaftor) to include children aged two to five years old.
Previous Authorisation: Before this announcement, these medicines were already authorised for treating cystic fibrosis in patients aged six years and older with a common gene mutation.
Treatment Purpose: Kaftrio and Kalydeco are used as long-term treatments to manage cystic fibrosis symptoms in patients with at least one F508del mutation in the CFTR gene—the most common mutation causing the condition.
Mechanism of Action: The active ingredients in these treatments work by interacting with abnormal CFTR proteins, enhancing chloride movement in and out of cells, which is impaired in people with cystic fibrosis.
Administration: The medicines are provided as granules mixed with soft food for immediate consumption with a fat-containing meal or snack, with doses varying depending on the child's weight.
Clinical Evidence: The extension of authorisation to younger children is backed by evidence from clinical studies in older patients and a 24-week phase 3 clinical study in 75 patients aged two to five.
Study Findings: The study showed that the treatment was safe and well-tolerated in the young participants, with a safety profile consistent with older age groups. The treatment effectively reduced sweat chloride concentrations, which is comparable to the effects seen in older patients.
Monitoring: The MHRA will continue to monitor the safety and effectiveness of the treatments and encourage reporting of any side effects through the Yellow Card scheme.
I will provide links below to learn more.
Unfinished Victories
Call your loved ones, pop the champagne, and give your little CFers an extra big squeeze tonight. Celebrate them, and as you look into their eyes, tell them that today is a truly wonderful day. If they respond to the medication as most often do, they may never fully realise its significance. The hope is that they will avoid a life of declining health, and so for them, with hope, normal life will be exactly that; normal.
One day, on some level they'll grasp it, and I’m sure they’ll be filled with nothing but respect for the unwavering love, care, and guidance you've provided as parents.
But as the celebrations wind down, the champagne flutes are tucked away, and everyday life kicks back in—a life hopefully far less consumed by CF—take a moment to make a promise to yourselves not to forget those still waiting. Remember those awaiting news from NICE, and think of the countless individuals with CF worldwide who may never gain access unless we continue to put pressure on Vertex Pharmaceuticals.
In our activism, we must also remember those not eligible for CFTR modulators. Keep them in your hearts, minds and lungs. Make a promise to support them, share their posts and their stories, and continue your fundraising efforts. We may still need to pick up our placards and fight, so please remain within this community and continue to offer your support as if your loved ones’ lives depend upon it.
You've earned this moment, cherish it and cue the music:
Marc x
Useful Links
MHRA Announcement: https://www.gov.uk/government/news/cystic-fibrosis-drugs-kaftrio-and-kalydeco-licensed-for-patients-aged-two-to-five-years-old
Yellow Card Scheme: https://yellowcard.mhra.gov.uk/
Sign the global access petition: https://actionnetwork.org/forms/cf-drugs-for-everyone-everywhere-now/