The U.S. Food and Drug Administration announced on Friday, Nov. 13 that it granted approval to Baxalta drug Adynovate for the treatment of patients with Hemophilia A. The Antihemophilic Factor and PEGylated drug was given the green light for affected individuals aged 12 years old and above.
The drug has been developed into a modified version such that it can last longer in the blood and possibly necessitate lesser frequency of administration compared to unmodified Antihemophilic Factor when injected to decrease bleeding episodes.
FDA approved the drug to be administered on an "as needed" basis to treat and control bleeding, as well as lessen bleeding frequencies among patients diagnosed with Hemophilia A.
The components of Adynovate include the complete range of Coagulation Factor VIII molecule connected with polyethylene glycol (PEGylated). The connection between these molecules is the secret to the long-lasting stay of the drug in the blood of patients.
A total of 137 adults and adolescent patients were involved in the clinical trial to test the safety and efficacy of Adynovate. The tests entailed comparisons of conventional preventive treatment modalities to on-demand or "as needed" therapy.
The results of the clinical trial showed that Adynovate was efficient in decreasing the frequency of bleeding episodes during routine care, as well as in treating and controlling bleeding. "No safety concerns were identified during the trial," the FDA press release reads.
Hemophilia A is an inherited disorder characterized by impaired blood-clotting mechanisms. Most commonly prevalent in males, the sex-linked disorder is caused by faults in the Factor VIII gene.
The Centers for Disease Control and Prevention (CDC) reported that Hemophilia A is present in about one in every 5,000 male births in the United States alone.
The signs and symptoms of the disorder include intermittent episodes of grave bleeding that most typically occurs in the joints. As a result, the joints can possibly undergo severe impairment.
Karen Midthun, director of the agency's Center for Biologics Evaluation and Research said that the green light given to Adynovate gives an essential therapeutic choice for patients with Hemophilia A and decreases the injections of Factor VIII needed to prevent bleeding.
Together with California-based Baxalta, which spun off Baxter earlier in 2015, Nektar Therapeutics Inc. also helps in the manufacture of Adynovate.
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