Pediatric Study - Results

No Factor VIII inhibitors were detected during the XTEND-Kids study^1*

Primary endpoint: Incidence of neutralizing antibodies to Factor VIII²

^* Defined as an inhibitor of ≥0.6 BU/mL and confirmed by a second test result from a separate sample drawn 2 to 4 weeks following the date of the original sample.²

Effective bleed protection with a once-weekly infusion¹

In the XTEND-Kids trial:

ALTUVIIIO demonstrated proven bleed protection in children <12 years (n=72)*

^* Based on treated bleeds.

Most patients experienced zero bleeds when treated with ALTUVIIIO prophylaxis¹

In the XTEND-Kids trial^*:

^* Based on treated bleeds.

Effective on-demand bleed management with only 1 infusion¹

Treatment of bleeding episodes in the XTEND-Kids trial^1,2

95.3% of bleeds* resolved with 1 infusion (n=41/43)

^* Percentage based on the number of treated bleeding episodes in the sensitivity analysis set, which excluded two patients.^1,2

Excellent perioperative response across both XTEND-1 and XTEND-Kids trials¹

The hemostatic response of ALTUVIIIO was deemed “Excellent/Good*” by the investigator surgeon in:

^* Hemostatic response during major surgery was assessed using the International Society on Thrombosis and Haemostasis (ISTH) 4-point response scale: excellent, good, moderate, or poor/none. Based on pooled data from XTEND-1 and XTEND-Kids.^1
† Defined as any surgical procedure, either elective or emergent, that usually involves general anesthesia and/or respiratory assistance, in which a major body cavity is penetrated and exposed, or a substantial impairment of physical or physiological functions is produced. Major surgeries include major orthopedic procedures such as arthroplasties; joint replacements of the knee, hip, and elbow; joint revisions; and ankle fusions. Other major surgeries include molar-tooth extractions, dental restoration and tooth extraction, circumcision, and rhinoplasty/mentoplasty.^1,2
‡ Median preoperative dose per surgery was 50 IU/kg (range: 12.7-61.9).¹