The supplemental BLA would expand Coagadex’s prescribing information to include prophylactic and pediatric data.
Bio Products Laboratory Limited (BPL) has announced that the US Food and Drug Administration (FDA) has accepted for filing a supplemental Biologics License Application (BLA) for Coagadex® (Coagulation Factor X, Human).
Coagadex, a plasma-derived blood coagulation factor X concentrate, is intended for prophylactic treatment of hereditary factor X deficiency, as well as treatment in children under 12 years of age.
"BPL is dedicated to improving the lives of patients with bleeding and clotting disorders, and is extremely pleased that FDA has accepted our supplemental BLA with Priority Review," said Eric Wolford, PharmD, Vice President of Global Medical at BPL.
The supplemental BLA submission was based on results from the phase 3 TEN02 prospective study of Coagadex for prophylaxis of bleeding episodes in children under 12 years old with moderate to severe hereditary factor X deficiency.
The open-label, multicenter study included patients <12 years of age with moderate or severe hereditary factor X deficiency and either a history of severe bleeding or an F10 gene mutation resulting in a documented severe bleeding type.
Just 9 pediatric patients participated in this trial. The hereditary factor X deficiency is a rare disorder that affects approximately 300-600 patients in the US. These patients have a higher risk of bleeding and must be managed comparably to patients with hemophilia.
Researchers recommended a prophylactic dose of 40-50 IU/kg twice weekly, but dose and frequency were adjusted over the initial 6 weeks of the study to maintain factor X concentration levels ≥5 IU/dL. Investigators rated the overall efficacy of Coagadex as “excellent” for all study subjects.
The study reported a total of 28 treatment-emergent adverse events (TEAEs) were reported in 8 unique subjects, none of which were considered treatment-related. The majority of TEAEs (n = 26) were mild in severity and the remaining 2 were moderate. Pyrexia and nasopharyngitis were the most commonly reported TEAEs. Just 2 serious adverse events were reported, but the investigation did not consider either treatment related.
Coagadex was previously approved in 2015 for adults and children ages 12 and older with hereditary factor X deficiency for on-demand treatment and control of bleeding episodes, as well as perioperative management of bleeding in patients with mild hereditary factor X deficiency.
"The addition of prophylactic and pediatric data to the prescribing information would represent another key step to ensure patients receive appropriate treatment for hereditary factor X deficiency, a rare and serious condition," said Wolford.
Coagadex is the only FDA-approved treatment for hereditary factor X deficiency. The FDA has granted priority review for the Coagadex application, indicating an expedited 6-month timeline for reviewing and taking action instead of the usual 10 months.