Pegunigalsidase alfa

Pegunigalsidase alfa is a enzyme replacement therapy (ERT) developed for the treatment of Fabry disease, a rare genetic disorder. It is a form of the enzyme alpha-galactosidase A (α-Gal A) that has been modified by the addition of polyethylene glycol (PEG), a process known as PEGylation. This modification increases the stability and half-life of the enzyme, allowing it to remain in the body longer and provide a more sustained therapeutic effect.

Mechanism of Action

Fabry disease is caused by mutations in the GLA gene, which encodes the enzyme α-Gal A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3) in the body. When α-Gal A is deficient or absent, Gb3 accumulates in various tissues and organs, leading to the symptoms of Fabry disease.

Pegunigalsidase alfa works by replacing the deficient α-Gal A enzyme. The PEGylated enzyme is taken up by cells, where it breaks down accumulated Gb3. This can help to alleviate the symptoms of Fabry disease and slow the progression of the disease.

Clinical Trials and Approval

Pegunigalsidase alfa has undergone clinical trials to assess its safety and efficacy in treating Fabry disease. In a Phase 1/2 clinical trial, the therapy was found to be well-tolerated and resulted in a significant reduction in Gb3 levels in patients.

The therapy has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), which provides certain incentives for the development of treatments for rare diseases.

See Also

• Enzyme replacement therapy
• Fabry disease
• Orphan drug