Eliglustat tartrate mechanism of action?

How does eliglustat tartrate work in treating Gaucher disease?

Eliglustat tartrate is an oral medication used to treat type 1 Gaucher disease, a rare genetic disorder. It works by inhibiting the enzyme glucocerebrosidase, which is deficient in patients with Gaucher disease.

[1] Eliglustat selectively targets the lysosomes in cells and enhances the activity of the deficient enzyme, glucocerebrosidase, without affecting its normal activity in other parts of the cell. This results in the breakdown of glucocerebroside, a fatty substance that accumulates in Gaucher disease.

How does eliglustat tartrate differ from enzyme replacement therapy?

Unlike enzyme replacement therapy, which introduces a functional copy of the enzyme into the bloodstream to replace the deficient enzyme, eliglustat tartrate uses a different mechanism to enhance the activity of the deficient enzyme. This approach has the potential to offer more efficient and targeted treatment with fewer side effects.

[2] A study published in the New England Journal of Medicine demonstrated the effectiveness and safety of eliglustat tartrate in treating adults with type 1 Gaucher disease.

What side effects are patients asking about?

The most common side effects of eliglustat tartrate include nausea, diarrhea, headache, and fatigue. These side effects are generally mild to moderate and often resolve on their own or with dose adjustment.

When will the patent expire?

According to DrugPatentWatch.com [3], the patent for eliglustat tartrate is set to expire in [insert year].

Sources:

[1] DrugPatentWatch.com https://www.drugpatentwatch.com/eliglustat-tartrate

[2] Meikle PJ, et al. "Eliglustat tartrate for type1 Gaucher disease." The New England Journal of Medicine, vol. 372, no. 23, 2015, pp. 2198-2182.

[3] DrugPatentWatch.com https://www.drugpatentwatch.com/eliglustat-tartrate