The sun finally shines to those with bleeding hearts - pun intended.
Patients suffering from a genetic sex-linked, blood-clotting disorder can now hope for better days ahead with the approval of a new treatment.
A recently approved treatment by the US Food and Drug Administration has been made available to adults and children who have Hemophilia B. The treatment, called Alprolix, has been considered as the first recombinant, DNA-derived therapy for the rare disorder.
"The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B," said Dr. Karen Midthun, FDA's Center for Biologics Evaluation and Research director.
The new Hemophilia treatment has been approved on March 28. Its approval is the first important development for the said disorder treatment in over 17 years. The FDA gave an orphan-drug designation for its use, for the reason that it's intended for the treatment of an uncommon condition or disease.
"The FDA approval of ALPROLIX is a significant milestone for the hemophilia B community, and represents an important first step in our commitment to transform the care of people with hemophilia," said Dr. George A. Scangos, Biogen Idec.'s chief executive officer.
National Hemophilia Foundation's chief executive officer Val Bias expressed the same sentiment on the said approval.
"The hemophilia community is excited about new therapy options for the prophylactic management of hemophilia B. This approval is a significant step forward because it provides an important new option and expanded choice for the hemophilia B community," said Bias.
According to the FDA, the treatment will help control and prevent episodes of bleeding, manage bleeding in surgical procedures as well as reduce or prevent frequency of bleeding episodes. Said therapy has a prolonged circulation in the patient's body.
"The safety and efficacy of Alprolix were evaluated in a multi-center clinical trial that compared each of two prophylactic treatment regimens to on-demand treatment. A total of 123 individuals with severe Hemophilia B, ages 12-71, were followed for up to a year and a half. The studies demonstrated the effectiveness of Alprolix in the prevention and treatment of bleeding episodes and during perioperative management of patients undergoing a surgical procedure. No safety concerns were identified in this trial," explains the FDA.
Hemophilia B mostly affects males. Its cause points to the defects in Factor IX gene. In the United States alone, the disorder affects around 3,300 people. Effects of the disorder include physical disability as well as repeated episodes of potentially serious bleeding, primarily into the joints, which can be damaged by the bleeding.
Biogen Idec, Inc. partnered with the Swedish Orphan Biovitrum AB to develop the Alprolix treatment.
