First-in-class ALTUVIIIO: XTEND-1 and
XTEND-Kids clinical trials

In previously treated patients (PTPs)
who had received Factor VIII replacement1,2*

XTEND-1 PIVOTAL TRIAL

Graph showing the trial design for the 52-week XTEND-1 Clinical Trial

The Prior Factor
VIII Prophylaxis
Group included the
first
female patient
with severe
hemophilia A in a
pivotal
Factor VIII
clinical trial1,3-17‡

XTEND-1 Study Design1,2:

A multicenter, open-label, nonrandomized trial in adults and adolescents aged ≥12 years; 158 males and 1 female) with severe hemophilia A, including an intrapatient comparison in patients who had participated in a prospective observational study prior to enrollment in XTEND-1 study.

XTEND-Kids:
Patients <12 years of
age were
enrolled in a pediatric trial1,2

XTEND-Kids Study Design:

The pediatric study enrolled 67 male PTPs <12 years of age with severe hemophilia A (31 subjects were 1 to 5 years of age; 36 were aged 6 to 11 years) at data cut-off. Of the 67 enrolled subjects, all received at least 1 dose of ALTUVIIIO.

*Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived Factor VIII or cryoprecipitate for at least 150 exposure days for patients aged 6 years and older, and over 50 exposure days for patients aged <6 years.1

†Efficacy of prophylaxis was evaluated in patients with at least 26 weeks of exposure to ALTUVIIIO (n=128).2

‡Pivotal trials for Recombinate™ (Antihemophilic Factor) did not present demographic data based on sex.17

XTEND-1 ENDPOINT

Primary endpoint1,2

Mean annualized bleed rate (ABR)

Mean annualized bleed rate (ABR)

Secondary endpoints1,2

Blood droplet

Intrapatient ABR comparison
(key secondary endpoint)

Intrapatient ABR comparison
(key secondary endpoint)

Doctor

Perioperative management

Perioperative management

Syringe

Treatment of bleeds

Treatment of bleeds

Physical health

Quality of life (physical
health and pain)

Quality of life (physical
health and pain)

Knee joint

Joint health outcomes

Joint health outcomes

Bar graph

Pharmacokinetics

Pharmacokinetics

Medal

Safety and tolerability

Safety and tolerability

XTEND-1: A large
group of 159
diverse
patients
were studied1,2

XTEND-1 trial design infographic displaying patient population information

See how ALTUVIIIO
performed in XTEND-1

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

EXPLORE EFFICACY RESULTS

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.

IU=international unit.

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.