Clinical trials are important in developing a better understanding of certain diseases or conditions, as well as how to treat them. According to HCP Live, researchers evaluated PRX-102 (pegunigalsidase alfa), a therapeutic option for Fabry disease, within the Phase 3 BRIGHT clinical trial. Ultimately, the trial met its endpoints, highlighting the benefits of PRX-102 for this condition.
To begin, let’s take a look at what PRX-102 is. According to Protalix Biotherapeutics, PRX-102 is:
designed to be a plant cell culture-expressed, and a chemically modified version of, the recombinant alpha-Galactosidase-A protein. Protein sub-units are covalently bound via chemical cross-linking using PEG chains, resulting in a more active and stable molecule compared to the current available versions of the molecule as seen in preclinical models.
It is delivered via intravenous infusions. During the trial, researchers sought to uncover the safety, efficacy, tolerability, and pharmacokinetics of PRX-102. Altogether, 30 adult patients enrolled. These patients received a 2mg/kg infusion every four weeks for a total of 14 infusions. Findings include:
- PRX-102 was effective in stabilizing plasma lyso-Gb3 and therefore stabilizing patient conditions.
- The treatment was relatively safe and well-tolerated. While 30% of patients experienced some sort of adverse reaction, these were mild to moderate in nature.
About Fabry Disease
Fabry disease is an inherited lysosomal storage disorder caused by GLA gene mutations. Normally, GLA encodes for the production of alpha-galactosidase A, an enzyme in lysosomes which helps to break down globotriaosylceramide, a fatty substance. In Fabry disease, the mutations prevent the body from breaking down globotriaosylceramide; as it builds up in cells, it causes damage and health issues. An estimated 1 in every 40,000-60,000 males is born with Fabry disease. In the past, many believed that females were more likely to be asymptomatic, carriers, or have mild symptoms. However, more recent research suggests that females with Fabry disease may experience moderate or even severe symptoms. Ultimately, more research is needed to understand how Fabry disease affects female patients.
Potential symptoms associated with Fabry disease include:
- Hearing loss
- Corneal opacity, causing dull and clouded vision
- Gastrointestinal issues, such as stomach pain or bowel movements right after eating
- Acroparesthesias (episodes of pain affecting the hands and feet)
- Hyperhidrosis (decreased ability to sweat)
- Angiokeratomas (small, dark bundles of red spots on the skin)
- Tinnitus (roaring/ringing in the ears)
- Heart attack
- Acute kidney damage
Learn more about Fabry disease.