Chiesi UK and Ireland has announced that the MHRA has approved an additional 2 mg/kg body weight every-four-week dosing regimen for pegunigalsidase alfa for Fabry adults with an enzyme replacement therapy (ERT).
The decision follows on from a positive European Medicines Agency (EMA) decision in March of this year.
Pegunigalsidase alfa is a long-term ERT for adult patients with a confirmed diagnosis of Fabry disease (alpha-galactosidase deficiency), a rare disease affecting approximately 1 in 40,000 people in the UK.
It was originally approved by the MHRA in August 2023, with a dosing regimen of 1 mg/kg body weight administered every two weeks.
The approval of the additional dosing regimen means that eligible UK adult Fabry patients would require half as many infusions, from approximately 26 per year to 13.
Derralynn Hughes, Professor of Experimental Haematology, Director of Research and Innovation and Co-Clinical Director of the NCL Cancer Alliance, said: "For many people living with Fabry disease, regular fortnightly infusions can have a significant impact on day-to-day life, affecting work, family routines and overall quality of life."
The patient community consistently highlights the need for treatment approaches that help reduce this burden and we look forward to being able to offer infusions every four weeks to those who are stable on their current ERT, with appropriate monitoring.
Chiesi added that the MHRA approval was based on results from an open-label, switch-over study, BRIGHT (formally PB-102-F50).
Bob Stevens, Group CEO of the MPS Society and CEO and Chair of Rare Disease Research Partners, added: "People living with Fabry disease plan their lives around treatment."
"The possibility of attending infusions once every four weeks instead of two would offer greater flexibility and may help ease some of the practical challenges that come with long-term treatment."
David Garzón, Senior Director, Rare Diseases, UK and Ireland, said: “We are pleased to receive MHRA approval for the additional dosing option for pegunigalsidase alfa in adults stable with an ERT."
This is a positive step forward in improving the quality of life for patients and their families.
Those eligible in the UK may be prescribed the new dosing regimen of pegunigalsidase alfa from the date of approval, if their healthcare professional determines it is appropriate.