Pegunigalsidase alfa is recommended, within its marketing authorisation, as an option for treating Fabry disease (also known as alpha-galactosidase deficiency) in adults. It is recommended only if the company provides it according to the commercial arrangement.
Why the committee made these recommendations
Usual treatment for Fabry disease is migalastat or enzyme replacement therapy (ERT) with agalsidase alfa or agalsidase beta. Pegunigalsidase alfa is another ERT.
Clinical trial evidence shows that pegunigalsidase alfa works as well as agalsidase beta. There is no direct clinical trial evidence comparing pegunigalsidase alfa with agalsidase alfa or migalastat. But, clinical experts advised that pegunigalsidase alfa is also likely to work as well as these 2 treatments.
Economic evidence suggests that pegunigalsidase alfa is cost saving when compared with the other ERTs and migalastat. So, it is recommended.