According to a story from PMLive, Leadiant Biosciences of Italy recently announced that its drug Revcovi has officially received approval from the US Food and Drug Administration. Revcovi was approved for the metabolic disorder adenosine deaminase deficiency, a rare disease that causes immunodeficiency. This is a new recombinant form of enzyme replacement that will supplement Leadiant’s currently available animal derived enzyme replacement therapy called Adagen.
About Adenosine Deaminase Deficiency
Adenosine deaminase deficiency is a rare metabolic disorder that is characterized by immunodeficiency. About 15 percent of severe combined immunodeficiency cases are linked to adenosine deaminase deficiency. The disorder leads to the accumulation of deoxyadenosine in the body of the patient. This buildup ultimately prevents immune cells such as B-cells and T-cells from dividing. In addition, lymphocytes are unable to mature. The symptoms of the disorder can appear at almost any time of life, from infancy to adulthood. The disease is caused by a defective gene located on chromosome 20. Symptoms of adenosine deaminase deficiency include slowed growth, developmental delays, pneumonia, skin rashes, and chronic diarrhea. This disorder was first discovered in 1972. There are limited treatment options available, which include bone marrow transplant, enzyme replacement therapy, and stem cell gene therapy. To learn more about adenosine deaminase deficiency, click here.
Adenosine deaminase deficiency can ultimately be lethal without treatment, and the appearance of Leadiant’s Adagen onto the scene was the first viable treatment alternative for patients aside from bone marrow transplant. The approval of Revcovi is the result of data from recent clinical trials which demonstrated the new therapy’s effectiveness. Revcovi was able to increase adenosine deaminase activity, improve lymphocyte counts, and reduce the concentration of toxic metabolic substances.
The new approval is much needed good news for Leadiant, which has faced criticism for raising the price of one of its other rare disease drugs for no apparent reason. In fact, the company is currently facing legal action over the controversy.
Regardless of the company’s other issues, the approval of Revcovi is a major boost for patients with adenosine deaminase deficiency and provides another valuable treatment option for this rare disease patient community.