Elfabrio receives vital MHRA authorisation

The therapy involves long-term enzyme replacement therapy for aduls with Fabry disease

Chiesi has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for Elfabrio across Britain.

Also known as pegunigalsidase alfa, the treatment involves long-term enzyme replacement therapy (ERT) among adult patients with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).

Pegunigalsidase alfa is produced in plant cells using recombinant DNA technology and the treatment remains the only PEGylated ERT for Fabry disease.

The MHRA verdict is based on an overall positive benefit/risk balance of pegunigalsidase alfa in the claimed indication, as stated in the European Medicines Agency’s assessment report.

The clinical development programme for the therapy consisted of 142 patients with Fabry disease, of which 112 received pegunigalsidase alfa every other week. The research took place across several different studies and demonstrated that the therapy is generally well tolerated, with the most common adverse reactions being infusion-related reactions, followed by asthenia and hypersensitivity.

It is understood that around 1,150 people across England live with symptomatic Fabry disease – a rare, progressive, X-linked inherited lysosomal storage disorder, triggered by a genetic mutation. The condition ultimately leads to an inherited deficiency of enzyme α-galactosidase that is normally responsible for the breakdown of globotriaosylceramide.

Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases at Chiesi UK&I, was optimistic about the verdict, saying that the “MHRA authorisation for pegunigalsidase alfa brings an additional licensed treatment option for Fabry patients across Great Britain”.

He added: “As part of our goal to ensure equal access to innovative therapies for people living with rare diseases, we are working closely with health technology appraisal agencies to ensure that all eligible patients can access this new treatment as soon as possible.”