Positive results of the Kids A-LONG phase 3 clinical study that evaluated the safety and efficacy of Eloctate, an investigational recombinant factor VIII Fc fusion protein product candidate, in children with severe haemophilia A, were presented.
Eloctate was generally well-tolerated and no inhibitors ( neutralising antibodies that may interfere with the activity of the therapy ) were detected.
Efficacy analyses showed twice-weekly prophylactic dosing with Eloctate maintained low bleeding rates in children.
Kids A-LONG is the first study to evaluate a long-lasting, investigational haemophilia therapy in children under 12 years old.
Eloctate was developed using a process called Fc fusion and is designed to prolong the circulation of infused clotting factor in the body.
Kids A-LONG investigated the safety, efficacy and pharmacokinetics ( measurement of the presence of the drug in a person's body over time ) of Eloctate in previously treated children under 12 years old with severe haemophilia A.
The primary endpoint of the study was to evaluate the frequency of inhibitors ( neutralising antibodies ) development and none were detected. Secondary endpoints included the overall and spontaneous annualised bleeding rates ( ABR ), or projected number of yearly bleeding episodes, and the number of injections used to prevent and treat bleeding episodes.
In the study, the relative increase in half-life in children with severe haemophilia A was consistent with the 1.5-fold increase in half-life seen in the A-LONG study of adults and adolescents.
Children treated prophylactically with Eloctate had an overall median ABR of 2.0 and a median ABR for spontaneous bleeds of 0.0.
Forty-six per cent of participants in the study experienced zero bleeding episodes.
Overall, ninety three per cent of bleeding episodes were controlled by one to two injections of Eloctate.
Kids A-LONG was a global, open-label, multi-centre study involving 71 boys with severe haemophilia A ( factor VIII activity less than 1 IU per dL, or 1% ) with at least 50 prior exposure days to factor VIII therapies.
The study was conducted at 23 haemophilia treatment Centres in eight countries.
Overall, 67 participants ( 94% ) completed the study ( 33 under six years old and 34 six to 11 years old ).
The average time participants spent in the study was 25 weeks and 61 participants received Eloctate injections on at least 50 separate days ( exposure days ) to assess inhibitor development.
All study participants were to be initially treated with twice-weekly prophylactic injections of Eloctate ( 25 IU/kg day 1, 50 IU/kg day 4 ). Study investigators could adjust the dose or interval based on individual response. Approximately ninety per cent of study participants were on twice-weekly dosing at the end of the study.
Eloctate was generally well-tolerated. No inhibitors to Eloctate were detected and no cases of serious allergic reactions were reported in any participants, all of whom switched from commercially available factor VIII products.
No serious adverse events were assessed to be related to drug by the investigator.
Two non-serious events, rash and myalgia ( muscle pain ), were considered related to Eloctate treatment and were reported in one participant each.
No participant discontinued the study due to an adverse event after receiving Eloctate.
The pattern of treatment-emergent adverse events reported was typical of the population studied and generally consistent with results seen in adolescents and adults in the A-LONG study. ( Xagena )
Source: Swedish Orphan Biovitrum AB ( SOBI ), 2014