NICE recommends new Fabry disease treatment

The National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Elfabrio^® (pegunigalsidase alfa) for Fabry disease (alpha-galactosidase deficiency) in adults.

A new Fabry disease treatment

The agreement made in the NICE’s Final Draft Guidance means there is now a “new treatment option for people living with Fabry disease across England,” according to Dr Kamran Iqbal, Head of Medical Affairs for Global Rare Diseases at Chiesi UK&I. “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.”

Pegunigalsidase alfa is a novel enzyme replacement therapy (ERT) administered via intravenous infusion every two weeks and delivers a modified version of the enzyme α-galactosidase A.

Data highlighted by Chiesi noted that the efficacy and safety profile of pegunigalsidase alfa was drawn from the outcomes of a clinical trials programme. Overall, it evaluated 142 patients with Fabry disease (94 males and 48 females). There were 112 patients who received pegunigalsidase alfa 1mg/kg every other week.

Positive regulatory activity for pegunigalsidase alfa in 2023

In February 2023, the Committee for Medicinal Products for Human Use (CHMP) recommended the first pegylated enzyme for Fabry disease, which is a rare condition. The EMA’s human medicines committee reviewed Phase III clinical data, which combined, represented over 400 years of exposure to PRX–102 (pegunigalsidase alfa).

Several months later, in May, pegunigalsidase alfa was given a marketing authorisation by the European Commission (EC). At the time, Dror Bashan, Protalix’s President and Chief Executive Officer declared that the EC’s approval of the medicine was a “significant milestone” for patients with disease.

At present, pegunigalsidase alfa is approved in the EU, Northern Ireland, and Great Britain for long-term ERT in adult patients who have been confirmed to have the disorder. The treatment is also approved in the US as a therapy for confirmed Fabry disease in adults.