FDA has approved Idelvion, the first coagulation factor-albumin fusion protein to treat patients with hemophilia B
The FDA ( Food and Drug Administration ) has approved Idelvion, coagulation factor IX ( recombinant ), albumin fusion protein, for use in children and adults with hemophilia B.
Idelvion is the first coagulation factor-albumin fusion protein product to be approved, and the second factor IX fusion protein product approved in the U.S. that is modified to last longer in the blood.
Idelvion is used to replace factor IX, a naturally occurring clotting factor that is missing ( functionally deficient ) or defective in people with hemophilia B ( also called congenital factor IX deficiency or Christmas disease ).
Idelvion is produced by recombinant DNA technology linking factor IX to albumin, a protein found in blood, which accounts for the product lasting longer when given intravenously.
Idelvion is indicated for on-demand ( as needed ) control and prevention of bleeding episodes, management of bleeding following surgery ( perioperative ) and as a routine preventative ( prophylaxis ) measure to reduce the frequency of bleeding episodes.
Idelvion potentially requires less frequent injections than unmodified factor IX when used for prevention.
The safety and efficacy of Idelvion were evaluated in two multicenter studies, which included a total of 90 adult and pediatric patients with hemophilia B between 1 and 61 years of age.
Idelvion was demonstrated to be effective in controlling bleeding episodes and in managing perioperative bleeding. Idelvion used as prophylaxis led to a significant reduction in the rate of spontaneous bleeding episodes per year despite less frequent infusions of Idelvion.
No safety concerns were identified in the studies. The most common side effect observed for Idelvion was headache.
According to the Centers for Disease Control and Prevention ( CDC ), hemophilia B is a rare inherited bleeding disorder that prevents blood from clotting normally. The disorder primarily affects males and, rarely, females.
People with hemophilia B can experience repeated episodes of potentially serious bleeding, mainly into the joints, which can be damaged by the bleeding. ( Xagena )
Source: FDA; 2016
Hemophilia A: FDA has approved Adynovate, an antihemophilic factor PEGylated, for use in children and surgical settings
The FDA ( U.S. Food and Drug Administration ) has approved Adynovate [ antihemophilic factor ( recombinant), PEGylated ], an...
Once-weekly prophylactic dosing in children: Alprolix meets primary endpoint and achieves low bleeding rates
Positive top-line results of the Kids B-LONG phase 3 clinical study that evaluated the safety, efficacy and pharmacokinetics of Alprolix...
Clopidogrel ( Plavix ) is a platelet aggregation inhibitor indicated for conditions such as myocardial infarction, stroke or established peripheral...
The FDA ( Food and Drug Administration ) has approved Eloctate, antihemophilic factor ( recombinant ), Fc fusion protein, for...
Hemophilia with inhibitors: a trial evaluating efficacy and safety of BAY 86-6150 has been discontinued
A trial evaluating the efficacy and safety of BAY 86-6150 in people with hemophilia A and hemophilia B with inhibitors...
Severe hemophilia B and AAV-mediated gene transfer: longer follow-up and expansion of the high dose cohort
Researchers have previously reported the preliminary safety and efficacy data of a novel gene transfer approach in six subjects with...
Severe hemophilia A: recombinant and plasma-derived Factor VIII products conferred similar risks of inhibitor development
For previously untreated children with severe hemophilia A, it is unclear whether the type of factor VIII product administered and...
Cancers in patients with hemophilia: a retrospective study from the Italian Association of Hemophilia Centers
The increased life expectancy of the hemophilia population, primarily as a result of advances in factor replacement therapy, has enabled...
A team of researchers has concluded that people with bleeding disorders such as hemophilia remain at risk from emerging infectious...