Pharmaceutical Business review

Chiesi, Protalix’s PRX-102 secure FDA nod to treat Fabry disease

PRX-102 is indicated to treat Fabry disease in adult patients. Credit: ProteinBoxBot at English Wikipedia/ commons.wikimedia.org.

The latest move follows the receipt of marketing authorisation for PRX-102 from the European Commission (EC) for the same indication.

The PEGylated enzyme replacement therapy (ERT), PRX-102 is a recombinant human α‑Galactosidase‑A enzyme expressed in plant-cell culture that is designed for providing a long half-life.

The therapy was found to have an initial half-life of 78.9 ± 10.3 hours, in clinical trials.

Chiesi Global Rare Diseases head Giacomo Chiesi said: “While much progress has been made in the treatment of Fabry disease, there is still a need for new treatment options.

“We established Chiesi Global Rare Diseases to deliver innovative therapies and solutions for people affected by rare diseases.

“With the FDA approval of PRX-102, we can now offer people living with Fabry disease an alternative treatment option.”

PRX-102’s tolerability, efficacy, and safety has been evaluated in a comprehensive clinical development programme, which was conducted in over 140 patients, who have received follow up treatment for up to 7.5 years.

It has been investigated in ERT-experienced and ERT-naïve patients, including in a head-to-head trial.

The therapy showed non-inferior efficacy to agalsidase beta in controlling the estimated glomerular filtration rate (eGFR) decline.

Protalix BioTherapeutics president and CEO Dror Bashan said: “We are extremely pleased to receive FDA approval of PRX-102 for the treatment of adult patients with Fabry disease.

“This approval is a testament to the dedication of the Protalix and Chiesi teams to deliver this much needed new therapeutic option to patients in need.

“The totality of clinical data suggests that PRX-102 has the potential to be a long-lasting therapy.”