The therapy has been developed for the treatment of Fabry disease among adult patients

Chiesi has announced that the National Institute for Health and Care Excellence (NICE) has recommended the company’s Elfabrio treatment.

The therapy, also known as pegunigalsidase alfa, has been developed to treat Fabry disease among adult patients with a confirmed diagnosis.

Pegunigalsidase alfa is a novel enzyme replacement therapy delivered through intravenous infusion every two weeks and provides a modified version of the enzyme, α-galactosidase.

The safety and efficacy profile of pegunigalsidase alfa was studied using data from a clinical trials programme, which included 142 individuals with Fabry disease (94 males and 48 females). Of these, 112 received pegunigalsidase alfa 1mg/kg every other week.

This research subsequently demonstrated that the treatment was generally well tolerated, with the most prevalent adverse reactions being infusion-related reactions, reported by 6.3% of patients, followed by asthenia and hypersensitivity, reported each by 5.6% of patients.

Dr Kamran Iqbal, head of medical affairs, global rare diseases at Chiesi UK&I, was optimistic about the recommendation: “We are delighted that NICE has recommended pegunigalsidase alfa, bringing a new treatment option for people living with Fabry disease across England.”

He added: "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.”

Bob Stevens, group chief executive at MPS Society, reflected: “On behalf of our Fabry community, the MPS Society welcomes the decision by NICE to make available the treatment pegunigalsidase alfa to our community, broadening the treatment options for those affected by Fabry.”

He concluded: “For people living with Fabry, it is vital that they are supported in living the lives they want and are able to make informed decisions about their treatment.”

Fabry disease remains a rare genetic disease impacting on around 1,150 people across England. Those with Fabry disease do not produce enough of the aforementioned enzyme, alpha-galactosidase A, which is required to break down various fatty acids.