The Food and Drug Administration has approved Idelvion, the first hemophilia B therapy with up to 14-day dosing intervals, and Kovaltry, an unmodified, full-length factor VIII compound for the treatment of hemophilia A.
Idelvion is a novel long-acting recombinant albumin fusion protein administered intravenously. In clinical trials from the PROLONG-9FP clinical development program – including phase I through III open-label, multicenter studies – the biotherapeutic agent maintained factor IX activity levels above 5% over 14 days at a dose of 75 IU/kg, resulting in a median annualized spontaneous bleeding rate of zero in patients, according to a statement by Idelvion’s manufacturer, CSL Behring. That “reduces the monthly number of units needed for prophylaxis therapy,” the company noted.
Idelvion is indicated in children and adults with hemophilia B for routine prophylaxis, as well as for on-demand control and prevention of bleeding episodes. It is also indicated for the perioperative management of bleeding. With on-demand treatment, 94% of bleeds were controlled with one infusion, and 99% were controlled with one or two infusions.
Appropriate patients 12 years and older can go up to 14 days between infusions, according to CSL Behring. The most common adverse reaction in the clinical trials was headache.
Idelvion is expected to be available later this month.
“The approval of this long-acting recombinant factor IX therapy for hemophilia B is vital, as physicians need more options to help their patients effectively and safely manage their bleeding disorder,” said Elena Santagostino, M.D., Ph.D., of the University of Milan/IRCCS Maggiore Hospital, and lead investigator of PROLONG-9FP, in a statement. “This provides them with greater freedom from frequent infusions.”
The FDA approved Kovaltry for children and adults with hemophilia A based on the results of the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease) clinical trials. Kovaltry received approval in Europe and Canada earlier this year.
The LEOPOLD findings supported the approval for routine prophylaxis to reduce the frequency of bleeding episodes.
“In the LEOPOLD trials, Kovaltry reduced bleeding episodes in patients with hemophilia A when infused twice to three times per week with routine prophylaxis,” said Dr. Sanjay P. Ahuja, LEOPOLD investigator and director of the hemostasis and thrombosis center at University Hospitals Rainbow Babies & Children’s Hospital, Cleveland, in a statement by Kovaltry manufacturer Bayer. “Kovaltry may offer appropriate patients a twice-weekly prophylaxis dosing option.”
Dosing is 20-40 IU/kg of body weight 2-3 times per week in adolescents and adults, and 25-50 IU/kg of body weight 2-3 times per week or every other day in children aged 12 years or younger.
The most common adverse events associated with Kovaltry in the clinical trials were headache, fever, and pruritus.