The National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Elfabrio (pegunigalsidase alfa) as a long-term option for treating Fabry disease in adults in England and Wales.
The NICE committee concluded that the treatment would be an additional ERT for patients with and without an amenable mutation and was a relevant comparator to existing treatments.
Elfabrio is a novel enzyme replacement therapy (ERT) that is administered via intravenous infusion every two weeks. The treatment works to deliver a modified version of the enzyme a-galactosidase for those with a confirmed diagnosis of Fabry disease.
The recommendation was based on results from phase 3 clinical trials, which showed that Elfabrio was generally well tolerated and was similarly clinically effective and as tolerable as the current treatments, agalsidase alfa and migalastat, used in the NHS.
It was also a more cost-effective option when compared with other ERTs and migalastat.
The treatment is the first ERT recommended for routine NHS use by NICE.
Affecting approximately 1,150 people in England, Fabry disease, also known as deficiency of alpha-galactosidase, is a rare genetic disease that can lead to progressive damage to vital organs, including the heart, kidney and brain.
Bob Stevens, group chief executive of the MPS Society, said: “We welcome the decision by NICE to make available the treatment pegunigalsidase alfa to our community, broadening the treatment options for those affected by Fabry.”
Dr Kamran Iqbal, head of medical affairs, global rare diseases at Chiesi UK and Ireland, said: “We are delighted that NICE has recommended pegunigalsidase alfa, bringing a new treatment option for people living with Fabry disease across England.
“Fabry disease brings a multitude of complex symptoms and, since one therapy may not suit all, it is vital that patients have additional treatment options available to them.”
The recommendation follows the UK Medicines and Healthcare products Agency (MHRA) decision to grant Elfabrio marketing authorisation in the UK as a long-term ERT in adult patients with a confirmed diagnosis of Fabry disease.