Recently, the U.S. Food and Drug Administration approved Adzynema, the first recombinant protein therapy indicated for prophylactic (preventive) enzyme replacement therapy in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (CTTP). Is.
CTTP is a rare and life-threatening blood disorder that causes blood clots to form in small blood vessels, resulting in low platelet counts. According to the FDA, this hereditary disorder develops due to disease-causing mutations in the ADAMTS13 gene, which is responsible for the production of an enzyme that regulates blood clotting, also known as ADAMTS13.
In relation to the symptoms of this disease, symptoms such as bleeding in the skin or mucous membranes, confusion, fatigue, weakness, fever, headache, pallor, difficulty in breathing and high heart rate (more than 100 beats per minute) arise. Similarly, people with this condition can experience severe bleeding episodes, stroke, and damage to vital organs.
CTTP is estimated to affect less than one in a thousand people in the United States, and symptoms typically develop in infancy or early childhood. However, in some cases they may develop in adulthood and first appear during pregnancy.
It is important to treat this disease in time, because otherwise it can be fatal. Thus, the FDA states that this approach involves prophylactic plasma-based therapy to reduce the risk of clotting or bleeding by replacing missing or reduced ADAMTS13 enzymes for people with chronic diseases.
Features of Adzinma, the first recombinant protein treatment product
This product is a purified recombinant form of the above enzyme that acts by replacing the low levels of the deficient enzyme in patients with CTTP. For its treatment, administration of Adizenma, which should be administered intravenously once every two weeks, helps to reduce the risk of symptoms of this pathology.
With respect to product safety and efficacy, the FDA demonstrated that a global study was able to evaluate prophylactic and on-demand ERT with adenoma compared to plasma-based therapy in patients with CTTP.
To this end, its effectiveness was evaluated in 46 patients who were randomly selected to receive 6 months of treatment with this product or plasma-based therapy. They were then transitioned to the other treatment for six months and efficacy was demonstrated based on the incidence of thrombotic thrombocytopenic purpura events, TTP manifestations, as well as the need for supplemental doses.
“The efficacy of on-demand ERT was evaluated based on the proportion of acute TTP events that responded to adzinma in both the prophylactic group and the on-demand group during the study period. All acute and subacute TTP events resolved after treatment with adenoma or plasma-based therapies, notes the FDA.
Adzinma side effects
According to the FDA, the most common side effects associated with this product include headache, diarrhea, migraine, stomach pain, nausea, upper respiratory tract infection, dizziness, and vomiting. Among clinical studies, no adverse events such as allergic reactions were observed that could alter the patient’s health.
Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said the administration is committed to efforts to help facilitate the development and approval of safe and effective treatments for patients with chronic diseases. As such, the approval of the rare product “represents significant progress in the development of much-needed treatment options for patients affected by this potentially fatal disorder today.”