Proven bleed protection
with ALTUVIIIO1,2

Low ABRs in the Prior
Factor VIII
Prophylaxis Group

Intrapatient ABR comparison (n=78)
Vs Prior Factor VIII PROPHYLAXIS1,2

Bar graph displaying a 77% mean reduction in ABR in 78 patients on prestudy Factor VIII prophylaxis vs. ALTUVIIIO prophylaxis in the XTEND-1 trial
ABR† WITH ALTUVIIIO PROPHYLAXIS (n=128)1,2
0.0
Median ABR (Q1, Q3: 0.0, 1.0)
0.7
PRIMARY ENDPOINT
Mean ABR‡ (95% CI: 0.5-1.0)

*Based on treated bleeds. 1

†Data from prospective observational study. 2

‡Based on negative binomial model. 1

§Data based on interim analysis in subjects
with at least 26 weeks of exposure. 1
ABR=annualized bleed rate; Q1=25th percentile;
Q3=75th percentile.

In the XTEND-Kids
trial (n=23)1,2:

Routine prophylaxis with ALTUVIIIO resulted
in a mean ABR of 0.5 (95% CI: 0.2-1.3) and a
median ABR of 0 (Q1, Q3: 0.0-1.3).§

Patients who switched to ALTUVIIIO prophylaxis
experienced significant reductions in ABR1,2

Intrapatient ABR comparison (n=78)
VS PRIOR FACTOR VIII PROPHYLAXIS1,2

showing prior factor VIII prophylaxis and ALTUVIIIO prophylaxis treatment mean ABR from the XTEND-1 trial

Intrapatient ABR comparison (n=26)
PRIOR FACTOR VIII ON-DEMAND GROUP1,2

Bar graph displaying a 97% mean ABR reduction in 26 patients in the prior Factor VIII on-demand group in patients with 26 weeks of prophylaxis treatment with ALTUVIIIO vs mean ABR of 26 weeks with on-demand treatment with ALTUVIIIO in the XTEND-1 trial

*Data from prospective observational study. 2
†Based on treated bleeds. 1

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.2

*Data from prospective observational study. 2

†Based on treated bleeds. 1

Proven joint bleed protection
and improved joint health

PRIOR FACTOR VIII PROPHYLAXIS GROUP1*
(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
PRIOR Factor VIII ON-DEMAND GROUP1*
(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. 1

AjBR=annualized joint bleed rate.

IMPROVED TARGET JOINTS
AND JOINT HEALTH1,2

Hemophilia Joint Health Score (HJHS) showed improvement: −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

IMPROVED TARGET JOINTS
AND JOINT HEALTH1,2

100% of target joints resolved at 52 weeks (n=45)

Target joints
resolvedǂ

Hemophilia Joint Health Score (HJHS)
showed improvement: −1.5 (−2.7, −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

§Mean change in total score from baseline to Week 52.2

Most patients
experienced ZERO
bleeds
when
treated with
ALTUVIIIO prophylaxis

Patients with zero bleeds1

Prior Factor VIII
PROPHYLAXIS Group

Infographic displaying 64.1% of patients with zero bleeds over 52 weeks in the prior factor VIII prophylaxis group (n=82/128) and 76.9% of patients with zero bleeds in the 26 week prophylaxis phase in the prior factor VIII on-demand groups (n=20/26) in the XTEND-1 trial

Prior Factor VIII

On-Demand Group

Infographic displaying 64.1% of patients with zero bleeds over 52 weeks in the prior factor VIII prophylaxis group (n=82/128) and 76.9% of patients with zero bleeds in the 26 week prophylaxis phase in the prior factor VIII on-demand groups (n=20/26) in the XTEND-1 trial

Patients with zero
joint bleeds1

Prior Factor VIII

PROPHYLAXIS Group

Infographic displaying 71.9% of patients with zero joint bleeds over 52 weeks in the prior factor VIII prophylaxis group (n=92/128) and 80.8% of patients with zero joint bleeds in the 26 week prophylaxis phase in the prior factor VIII on-demand groups (n=21/26) in the XTEND-1 trial

Prior Factor VIII

On-Demand Group

Infographic displaying 71.9% of patients with zero joint bleeds over 52 weeks in the prior factor VIII prophylaxis group (n=92/128) and 80.8% of patients with zero joint bleeds in the 26 week prophylaxis phase in the prior factor VIII on-demand groups (n=21/26) in the XTEND-1 trial

*Based on treated bleeds. 1

Effective on-demand bleed management
with only 1 infusion1

Treatment of bleeding episodes in the
XTEND-1 trial

6.7% of bleeds occurring in the XTEND-1 trial were resolved with 1 infusion (n=350/362)

resolved
with

6.7% of bleeds occurring in the XTEND-1 trial were 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.2

ALTUVIIIO was
shown to provide
effective bleed

resolution and
perioperative
management

Hemostatic effect of ALTUVIIIO was rated “excellent” by the investigator or surgeon in 100% of major surgeries

Excellent perioperative response
in the XTEND-1 trial

  • In 100% of major surgeries (n=13/13),
    the hemostatic
    effect of ALTUVIIIO
    was rated
    as “excellent” by the
    investigator or surgeon†

Excellent perioperative
response in the
XTEND-1 trial

  • In 100% of major surgeries (n=13/13), the hemostatic effect of ALTUVIIIO was rated as “excellent” by the investigator or surgeon.†

†Of the 13 major surgeries, 12 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.1

Pain and
physical health scores
from
patient-reported outcomes

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

REDUCTION IN PAIN2

REDUCTION IN PAIN2

Mean change†: -0.2 (95%
CI: 0.02 to -0.0)2

REDUCTION OBSERVED IN
PROMIS PAIN INTENSITY SCORE2*

PROMIS Pain Intensity2*

Graph displaying pain intensity data from XTEND-1 trial in patients in prior FVIII prophylaxis group

Mean change†: -0.2 (95% CI: 0.02 to -0.0)2

improved
physical health2

improved physical health2

Mean change†: -6.7 (95%
CI: -10.1 to -3.4)2

HAEM-A-QOL Physical
Health Score
IN PATIENTS ≥17 YEARS OF AGE2†

HAEM-A-QOL PHYSICAL HEALTH SCORE
IN PATIENTS ≥17 YEARS OF AGE2†

Graph displaying physical health outcomes from XTEND-1 trial in patients in prior Factor VIII prophylaxis group

Mean change†: -6.7 (95% CI: -10.1 to -3.4)2

  • 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.

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.2

*Worst pain experienced during the last 7 days (1=no pain; 2=mild pain; 3=moderate pain; 4=severe pain; 5=very severe pain).2

†LS mean based on mixed-effect model with repeated measures (MMRM) with visit as fixed effect, and baseline score as a covariate.2

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 study2

ARM A:
PROPHYLAXIS GROUP2

Altuviio vs Prior Prophylaxis treatment

ARM B:
ON-DEMAND GROUP2

Altuviio vs Prior On-Demand treatment

Among the 142 participants who preferred treatment with ALTUVIIIO,
the most common reasons for preference were3:

Less frequent treatment

Less frequent treatment

81%
 (n=115)

Bleeds reduction

Bleeds reduction

Feel better protected

Feel better protected

69%
(n=98)

64%
(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 option2

Safety from the XTEND-1 Phase 3 trial

Find out more about the
trial with the
ALTUVIIIO
safety profile.

LEARN ABOUT SAFETY

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 is 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.

See full Prescribing Information.

Learn more about Sanofi’s commitment to fighting counterfeit drugs.

ABR=annualized bleed rate; AjBR=annualized joint bleed rate; CI=confidence interval; Haem-A-QoL= Haemophilia Quality of Life Questionnaire for Adults; HJHS=hemophilia joint-health score; Q1=25th percentile; Q3=75th percentile; ISTH=International Society of Thrombosis and Haemostasis; LS=least squares; PRO=patient-reported outcomes; PROMIS=Patient-Reported Outcomes Measurement Information System; QoL=quality of life.

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

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 is 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.

Please see full Prescribing Information.

Learn more about Sanofi’s commitment to fighting counterfeit drugs.

aPTT=activated partial thromboplastin time; PK=pharmacokinetics.