The use of pegunigalsidase alfa in the treatment of adults with Fabry disease (alpha-galactosidase deficiency) has been granted approval by the UK’s National Institute for Health and Care Excellence (NICE).
In its draft guidance, NICE recommended pegunigalsidase alfa (brand name Elfabrio®), which is manufactured by Chiesi, as a long-term treatment for adult patients with a confirmed diagnosis of Fabry disease with and without an amenable mutation. It is based on the terms of a confidential commercial agreement.
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.
It was found to be similarly clinically effective and as tolerable as the treatments for Fabry disease used currently in the NHS, and costs less. This approval makes it the first ERT treatment recommended for routine NHS use by NICE.
Commenting on the approval, Dr Kamran Iqbal, head of medical affairs, global rare diseases, Chiesi UK&I said: ‘We are delighted that NICE has recommended pegunigalsidase alfa, bringing a new treatment option for people living with Fabry disease across England. 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.‘
Fabry disease is a rare genetic disorder affecting approximately one in 49,000 people in England and is closely related to mucopolysaccharide (MPS) diseases. It results from a lack of the enzyme, alpha-galactosidase A, which degrades certain fatty acids. With insufficient amounts of the enzyme available, a build-up of these fatty acids occurs and causes progressive damage to vital organs including the heart, kidney and brain.
Patients with Fabry disease experience a range of symptoms which include short-term severe pain (lasting for minutes to days) or a burning sensation starting at the extremities and spreading throughout the body, which is referred to as a ‘Fabry crisis’. In addition, patients can have gastrointestinal symptoms such as diarrhoea, nausea and abdominal pain, headaches, hypohidrosis and vertigo.
Bob Stevens, group chief executive at the MPS Society, added: ‘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.
‘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.‘