Altuviiio logo

Menu

expand_more

Adult & Adolescent Study - Efficacy


Proven bleed protection with ALTUVIIIO1

Low ABRs in the Prior Factor VIII Prophylaxis Group1,2

ABR* with ALTUVIIIO prophylaxis (N=128)

img_efficacy_1.png

0.0 Median ABR 
(Q1, Q3: 0.0, 1.0)

img_efficacy_2.png

PRIMARY ENDPOINT
0.7 Mean ABR† 
(95% Cl: 0.5-1.0)

* Based on treated bleeds.2
Based on negative binomial model.2

Patients who switched to ALTUVIIIO prophylaxis experienced significant reductions in ABR2

Intrapatient ABR comparison (n=78) vs prior Factor VIII prophylaxis

Charts

* Data from prospective observational study.1
‡ Based on treated bleeds.1

Intrapatient ABR comparison (n=26) prior Factor VIII On-Demand Group1,2

charts

Mean number of bleeding episodes reported during the 12 months prior to the study was 35.7 (SD: 22.2) in the Prior Factor VIII On-Demand Group.1

* Based on treated bleeds.1

Proven joint bleed protection and improved joint health1,2

img_0.png

Prior Factor VIII Prophylaxis Group* (n=128)

0.0 Median AjBR (Q1, Q3: 0.0, 1.0)

Mean AjBR†: 0.5 (95% CI: 0.4, 0.7)
52 weeks of prophylaxis

img_0.png

Prior Factor VIII On-Demand Group* (n=26)

0.0 Median AjBR (Q1, Q3: 0.0, 0.0)

Mean AjBR†: 0.6 (95% CI: 0.3, 1.5)
26 weeks of prophylaxis

* Based on treated bleeds.1
† Based on negative binomial model.2

img_efficacy_3.jpg

Improved target joints and joint health1,2

Hemophilia Joint Health Score (HJHS) showed improvement1:

−1.5 (−2.7 to −0.4) points in subjects on prophylaxis at Week 52 vs baseline§

  • HJHS is a validated tool used to measure joint health. The open-label nature of the study may impact interpretation of the results

All patients with target joints at baseline (defined as ≥3 spontaneous bleeding episodes in a major joint which occurred in a consecutive 6-month period) achieved resolution of all target joints (45/45, 100%) with 12 months of prophylactic treatment with ALTUVIIIO (defined as ≤2 bleeding episodes in the target joint in 12 months).1,2
§ Mean change in total score from baseline to Week 52.1

Most patients experienced ZERO bleeds when treated with ALTUVIIIO2

Patients with zero bleeds*

img_Percentages.png

Prior Factor VIII Prophylaxis Group

64.1% of patients (n=82/128) had zero bleeds in 52 weeks

img_percentages 2.png

Prior Factor VIII On-Demand Group

76.9% of patients (n=20/26) had zero bleeds in 26-week prophylaxis phase

Patients with zero joint bleeds*

efficacy-chart-12@2x (1) 1.png

Prior Factor VIII Prophylaxis Group

71.9% of patients (n=92/128) had zero joint bleeds in 52 weeks

img_efficacy_percentage 4.png

Prior Factor VIII On-Demand Group

80.8% of patients (n=21/26) had zero joint bleeds in 26-week prophylaxis phase

* Based on treated bleeds.2

Effective on-demand bleed management with only 1 infusion2

Treatment of bleeding episodes in the XTEND-1 trial2

treatment of bleeding episodes in the XTEND-1 trial

96.7% of bleeds* resolved with 1 infusion (n=350/362)

* One 50 IU/kg infusion of ALTUVIIIO (350/362 bleeds). Substantial majority of bleeding events in XTEND-1 (74%, 268/362) occurred during on-demand treatment in the Prior Factor VIII On-Demand Group.1

ALTUVIIIO was shown to provide effective bleed resolution and perioperative management2

Excellent hemostatic effect in 100% of major surgeries

Excellent perioperative response in the XTEND-1 trial2

In 100% of major surgeries (n=12/12), the hemostatic effect of ALTUVIIIO was rated as “excellent” by the investigator or surgeon†

Of the 12 major surgeries, 11 surgeries required a single preoperative dose to maintain hemostasis during surgery; for 1 major surgery during routine prophylaxis, no preoperative loading dose was administered on the day of or before surgery.3

Pain and physical health scores from patient-reported outcomes

In patients on ALTUVIIIO prophylaxis who switched from prior prophylaxis in the XTEND-1 trial1

PROMIS pain intensity*

Chart 3

REDUCTION IN PAIN

Mean change†
-0.2 (95% CI: -0.4 to 0.0)

HAEM-A-QoL Physical-Health Score in patients ≥17 years of age1

charts 4

IMPROVED PHYSICAL HEALTH

Mean change†
-6.7 (95% CI: -10.1 to -3.4)

  • A lower score represents an overall improvement in these measures

XTEND-1 was a single-arm study, which may impact the interpretation of patient-reported outcome findings. The absence of a comparator arm may result in an under- or overestimation of treatment effect. Patient-reported nature of data may impact the reliability of findings. The PROMIS instrument has not been validated in hemophilia patients.1

Study design: Patient-reported outcomes of pain intensity and physical functioning were evaluated in patients receiving ALTUVIIIO prophylaxis in the prior Factor VIII prophylaxis group. The PROMIS Pain Intensity 3a instrument was used to assess pain, specifically, the first question that rates the worst pain experienced during the last 7 days. Physical functioning was assessed in patients ≥17 years old using the Physical-Health Score of Haem-A-QoL, which evaluated factors such as painful swellings, presence of joint pain, pain with movement, difficulty walking far, and needing more time to get ready.1

* Worst pain experienced during the last 7 days (1=no pain; 2=mild pain; 3=moderate pain; 4=severe pain; 5=very severe pain).1
† LS mean based on mixed-effect model with repeated measures (MMRM) with visit as fixed effect, and baseline score as a covariate.1

XTEND-1 treatment preference: participant-reported outcomes

Patient preference was evaluated using a 2-item questionnaire on perceived impact of treatment at the end of the study3

img_efficacy_90.png

Arm A: Prophylaxis Group

90% preferred ALTUVIIIO vs prior prophylaxis treatment (n=117/130)

img_efficacy_100.png

Arm B: On-Demand Group

100% preferred ALTUVIIIO vs prior on-demand treatment (n=25/25)

Among the 142 participants who preferred treatment with ALTUVIIIO, the most common reasons for preference were3:
Less frequent treatment81% (n=115)
Bleeds reduction69% (n=98)
Feel better protected64% (n=91)

XTEND-1 was an open-label, single-arm study, which may impact the interpretation of patient-reported outcome findings. Patient-reported nature of data may impact the reliability of findings. The treatment preference questionnaire used is not a validated instrument.

In XTEND-1, most participants indicated they preferred treatment with ALTUVIIIO vs their previous hemophilia treatment option3

icon_kids.png

Safety from the XTEND-1 Phase 3 trial

Find out more about the adult and adolescent trial with the ALTUVIIIO safety profile.

icon_charts_v2.png

See how ALTUVIIIO performed

ALTUVIIIO has efficacy data in the prophylaxis, on-demand, and perioperative settings for children.

Indication

ALTUVIIIO® [antihemophilic factor (recombinant), Fc-VWF-XTEN fusion protein-ehtl] is a von Willebrand Factor (VWF) independent recombinant DNA-derived, Factor VIII concentrate indicated for use in adults and children with hemophilia A (congenital factor VIII deficiency) for:

  • Routine prophylaxis to reduce the frequency of bleeding episodes
  • On-demand treatment & control of bleeding episodes
  • Perioperative management of bleeding
Limitation of Use

ALTUVIIIO is not indicated for the treatment of von Willebrand disease.

Important Safety Information
Contraindications

ALTUVIIIO is contraindicated in patients who have had severe hypersensitivity reactions, including anaphylaxis, to the product or its excipients.

Warnings and Precautions
  • Allergic-type hypersensitivity reactions, including anaphylaxis, may occur with ALTUVIIIO. Allergic-type hypersensitivity reactions were not reported in the clinical trials. Advise patients to discontinue use of ALTUVIIIO if hypersensitivity symptoms occur and contact a physician and/or seek immediate emergency care.
  • Formation of neutralizing antibodies (inhibitors) to Factor VIII are possible following administration of ALTUVIIIO. Neutralizing antibodies were not reported in the clinical trials. Monitor all patients for the development of Factor VIII inhibitors by appropriate clinical observations and laboratory tests.
  • If assessment of plasma Factor VIII activity is needed, it is recommended to use a validated one-stage clotting assay. The ALTUVIIIO Factor VIII activity level is overestimated by the chromogenic assay and a specific ellagic acid-based aPTT reagent in one-stage clotting assay by approximately 2.5-fold. If these assays are used, divide the result by 2.5 to approximate the patient's ALTUVIIIO Factor VIII activity level.
Adverse Reactions

The most common adverse reactions (>10% of subjects) reported in clinical trials were headache and arthralgia.

IndicationImportant Safety Information

ABR=annualized bleed rate; AjBR=annualized joint bleed rate; CI=confidence interval; FVIII=Factor VIII; Haem-A-QoL=Haemophilia Quality of Life Questionnaire for Adults; IU=international unit; LS=least squares; PROMIS=Patient-Reported Outcomes Measurement Information System; Q1=25th percentile; Q3=75th percentile; SD=standard deviation.

References: 1. von Drygalski A, et al. N Engl J Med. 2023;388(4):310-318. 2. ALTUVIIIO Prescribing Information. Bioverativ Therapeutics Inc. Waltham, MA. 3. Data on file, June 2022.

© 2024 Sanofi. All rights reserved. ALTUVIIIO, MyALTUVIIIO, and Sanofi are registered trademarks of Sanofi or an affiliate.

MAT-US-2403560-v1.0-05/2024 Last Updated: May 2024