Elfabrio soon available at low or no cost for Fabry patients in England

About 1,150 people in the country are estimated to have the disease

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

Share this article:

Share article via email
A bell labeled

The National Institute for Health and Care Excellence (NICE) in England has recommended that Elfabrio (pegunigalsidase alfa) be covered by the country’s national health service (NHS) and provided at low or no cost to adults with Fabry disease.

About 1,150 people in England are estimated to have Fabry, according to Chiesi Global Rare Diseases, which codeveloped the enzyme replacement therapy (ERT) with Protalix BioTherapeutics.

Formerly called PRX-102, Elfabrio was approved in the U.K. for long-term ERT in adults with Fabry disease in August, following similar approvals in the European Union and the U.S. in May.

“We are delighted that NICE has recommended [Elfabrio], bringing a new treatment option for people living with Fabry disease across England,” Kamran Iqbal, physician and head of medical affairs, global rare diseases, at Chiesi U.K. and Ireland, said in a company press release. “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.”

Recommended Reading
An illustrated banner showing a world map with dotted lines connecting different destinations for Susanna VanVickle's column,

Enzyme replacement therapy for Fabry is a gift and a challenge

Elfabrio and Fabry disease

Fabry disease is a rare genetic disorder wherein mutations in the GLA gene result in not enough alpha-galactosidase A (Gal A) enzyme being produced. Gal A is needed to break down certain fatty molecules and its deficiency causes those molecules to accumulate to toxic levels and disrupt the function of various organs, including the kidneys and heart.

ERT for Fabry delivers a lab-made version of the Gal A enzyme to the bloodstream, which is expected to lower tissue and organ damage and reduce symptoms.

Administered via an into-the-vein infusion once every two weeks, Elfabrio provides a plant-based, version of Gal A that’s designed to last longer in the bloodstream than standard ERT products.

Its approvals were backed by findings from three Phase 3 clinical trials: BRIDGE (NCT03018730), BALANCE  (NCT02795676), and BRIGHT (NCT03180840).

These studies tested Elfabrio against Fabrazyme (agalsidase beta), an approved ERT, or assessed its safety and effectiveness in patients previously on Fabrazyme or Replagal (agalsidase alfa), an ERT available in Europe, but not the U.S.

Elfabrio was well tolerated and preserved kidney function or slowed its decline to comparable levels to the other ERTs, results showed.

What the NHS listing means

Its listing on the NHS means Elfabrio will be available for free or with a small copay to eligible patients. NICE is responsible for drafting recommendations about which therapies will be listed and who will be eligible for them.

The committee analyzes a treatment’s potential benefits along with its cost, which is determined in negotiations between the NHS and the therapy’s developer. Terms of the commercial agreement between NHS England and Chiesi are confidential and the agreed price was not disclosed.

The drafted NICE guidelines are expected to be finalized and published on Oct. 4.

“On behalf of our Fabry community, the MPS Society welcomes the decision by NICE to make available the treatment [Elfabrio] to our community, broadening the treatment options for those affected by Fabry,” said Bob Stevens, group chief executive of the MPS Society. “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.”