Is Altuviiio approved by the FDA?

Yes, Altuviiio is FDA approved. The FDA granted approval to Altuviiio as a first-in-class, high-sustained factor VIII replacement therapy for hemophilia A patients on February 22, 2023. This approval was based on data from the Phase 3 XTEND-1 study (NCT04161495) and XTEND-Kids study (NCT04759131).

Indications:

Altuviiio is approved 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 and control of bleeding episodes
Perioperative management of bleeding

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

How Does Altuviiio Work?

Altuviiio is a recombinant or man-made factor VIII (FVIII) replacement therapy that is independent of von Willebrand factor (VWF). This means that Altuviiio has a longer half-life, lasting 3 to 4 times longer than other standard and extended half-life FVIII products. As a result, it simplifies the dosing schedule to only once a week. Altuviiio functions by replacing the missing coagulation factor VIII in hemophilia A patients, thus lowering their risk of bleeding.

Usage Instructions:

Altuviiio is administered as a once-weekly intravenous infusion. The dosage is tailored based on the patient's needs and the severity of their condition:

Routine Prophylaxis: Once weekly infusion
On-demand Treatment: Dosage depends on the severity of the bleeding episode; an initial single dose with additional doses every 2 to 3 days may be considered
Perioperative Management: Dosage depends on the type of surgery; an initial single dose with additional doses every 2 to 3 days may be considered if clinically necessary

Warnings and Precautions:

Hypersensitivity Reactions: Allergic reactions, including anaphylaxis, may occur. Patients should discontinue use and seek immediate medical attention if they experience symptoms like shortness of breath, wheezing, hives, chest tightness, hypotension, and itching.
Neutralizing Antibodies: The formation of neutralizing antibodies to FVIII is possible. If bleeding is not controlled or plasma Factor VIII levels do not increase as expected, testing for neutralizing antibodies should be performed.

Common Side Effects:

Headache
Arthralgia (joint pain)

Important Considerations:

Pregnancy: It is not known if Altuviiio can affect reproductive capacity or cause fetal harm. Women who are pregnant or planning to become pregnant should discuss potential risks with their healthcare provider.
Breastfeeding: It is not known if Altuviiio passes into breast milk or its effects on a breastfed infant. Women who are breastfeeding or planning to breastfeed should consult their healthcare provider.

Storage Instructions:

Prior to Reconstitution: Store in the original package to protect from light. Can be stored at 2°C to 8°C (36°F to 46°F) or at room temperature up to 30°C (86°F) for a single period of up to 6 months. Do not freeze.
After Reconstitution: The reconstituted product may be stored at room temperature, not exceeding 30°C (86°F), for up to 3 hours. Protect from direct sunlight. Discard any unused reconstituted solution after 3 hours.

Conclusion:

Altuviiio is an FDA-approved, once-weekly recombinant factor VIII replacement therapy for hemophilia A patients. Its extended half-life offers a simplified dosing schedule and effective management of bleeding episodes. Patients should follow their healthcare provider's instructions and be aware of potential side effects and necessary precautions to ensure safe and effective treatment.

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