The family of a Newry boy who fought for access to a new “life-changing” medication have hailed May 8 as a “very, very good day” upon learning that that very medication has been approved for use!
Jamie and Colleen Pentony – the parents of 12-year-old Alfie Pentony – have been campaigning for their son’s access to treatment since his diagnosis in 2016 with Duchenne Muscular Dystrophy.
For ten years, Alfie has been managing the inherited muscle wasting condition through bi-annual appointments at a private hospital in Newcastle, steroid medication and yearly visits to America for advanced specialist treatment.
However, in February 2024, a new trial drug called Givinostat was approved for UK use by Medicines and Healthcare products Regulatory Agency (MHRA) for all people with DMD aged 6 and older.
However, access to the drug had not been granted for Alfie and 12 others like him living in Northern Ireland.
One of the major stumbling blocks was that The National Institute for Health and Care Excellence (NICE) had not approved its use.
However, on May 8 good news was delivered to the Pentonys and all parents of children with DMD… givinostat has been approved.
Around 530 people in England will now benefit from access to the Duchenne muscular dystrophy treatment givinostat, following the successful negotiation of a commercial deal to make the medicine available on the NHS.
Givinostat will be funded immediately through the Innovative Medicines Fund and becomes the third treatment for the condition we’ve recommended. Significantly, it provides an option for people with DMD that can delay the progression of the disease irrespective of the type of DMD they have.
Evidence from clinical trials suggests that givinostat may increase the length of time people with DMD can walk by around five years on average compared with established clinical management, which includes treatment with corticosteroids and supportive care. However, because of limitations in the evidence, it is not yet known how much benefit givinostat provides beyond that.
Announcing her delight, Colleen took to social media, via the Fight for Alfie page, to say: “Today is an incredible day.
“After all of Alfie’s hard work, strength, determination, the tears, the battles, and the unwavering support from so many families fighting alongside us, NICE has finally approved givinostat for boys over six who are ambulant. We are absolutely overwhelmed with happiness and hope.
“For Alfie to now have access to this treatment, alongside the opportunity to now go back and continue receiving treatments in America, means more than words can ever describe. Nearly ten years ago we took on Duchenne, and our incredible boy is still walking, still running, still kicking a football and proving every single day just how strong he is.
“Today feels bigger than just one victory, it feels like hope for the future. Hope for more treatments, more breakthroughs, more time, more memories, and more milestones for all our Duchenne warriors and their families. The future is looking brighter than ever, and we will never stop believing in what is still to come.
“Today, we are also thinking of the families with boys no longer walking, and especially those who have lost their ability while hospitals had been arguing over EAP’s and NICE having been dragging their heels leaving it to late for them.
“Alfie Pentony, we are beyond proud of you. You inspire us every day with your courage, your fight, and your smile. Keep kicking Duchenne’s butt like the absolute superstar you are. Today is a very, very good day!”
