Voxzogo

About Vosoritide

• Voxzogo, containing vosoritide is a medication approved by the U.S. Food and Drug Administration for treating achondroplasia in children under 5 years, as of October 20, 2023. 
• Achondroplasia is a genetic condition characterized by severely short stature and disproportionate growth. Individuals with achondroplasia typically have a genetic mutation that results in the overactivity of the fibroblast growth factor receptor 3 gene, hindering normal bone growth. 
• Voxzogo works by binding to the natriuretic peptide receptor-B, reducing the activity of the growth regulation gene, and stimulating bone growth. 
• The medication received approval in the European Union in August 2021 and the United States in November 2021, being recognized by the FDA as a first-in-class medication.

Strength: 

The lyophilized powder in a single-dose vial for reconstitution is available in strengths of 0.4 mg, 0.56 mg, or 1.2 mg for injection.

Recommended Dosage:

• Voxzogo injection is administered through subcutaneous injections once daily, consistently at the same time each day. 
• Regularly assess growth and modify the dosage based on body weight. Cease administration permanently upon epiphyseal closure.
• If your child’s healthcare provider approves home administration by a caregiver, proper training on Voxzogo preparation and injection is necessary before attempting to administer it. The healthcare provider will specify the frequency of Voxzogo doses.
• In case of a missed dose, it can be given within 12 hours of the scheduled time; otherwise, skip the missed dose. Your child should consume a meal and fluids about an hour before injection. 
• If unsure about the injection timing, consult the healthcare provider. 
• Dosing is weight-dependent, determined by the healthcare provider, who will monitor your child’s growth and provide guidance on discontinuation if necessary. Cease administration if directed by the healthcare provider.

Important:

Ensure cleanliness by washing your hands thoroughly with soap and water. Be cautious not to place opened items on unclean surfaces. Before opening the packaging, confirm that the strength of Voxzogo matches your prescribed dosage. After removing from the refrigerator, allow the Voxzogo vial and prefilled diluent syringe to reach room temperature before mixing. Inspect the vial and supplies for damage or contamination, refraining from use if compromised. 

Check the expiration date on the carton, vial, and prefilled diluent syringe; do not use if expired. Before injection, ensure your child has consumed a meal and a glass of fluid (8 to 10 ounces) within an hour. Administer Voxzogo at the same time daily, without combining it with other medications. Use the mixed solution immediately, discarding it if left at room temperature for over 3 hours, and dispose of it in a sharps container. Avoid reusing supplies; discard the used vial after injection, even if Voxzogo remains.

Warnings & Precautions

• Do not consume the medicine if you are allergic to Voxzogo or any of the medicine’s ingredients. 
• Before administering Voxzogo to your child, it is essential to inform their healthcare provider of all their medical conditions, including any kidney problems. 
• There is a risk of transient decreases in blood pressure associated with the use of Voxzogo.

Common Voxzogo Side Effects:

• Injection site reactions (redness, itching, swelling, bruising, rash, hives, pain)
• High blood alkaline phosphatase (shown in blood tests)
• Vomiting
• Joint pain
• Decreased blood pressure (dizziness, fatigue, nausea)
• Stomachache
• Difficulty moving
• Dry skin
• Ear pain
• Feeling of movement
• Muscle pain or stiffness
• Pain in the joints
• Spinning sensation
• Unusual drowsiness
• Trouble sleeping
• Severe allergic reaction (hives, difficulty breathing, swelling)
• Severe low blood pressure (dizziness, faintness, lightheadedness)
• Blurred vision, confusion, cough, trouble sleeping, vomiting (signs of posterior uveal melanocytosis)

Use in Specific Population

• Limited information is available regarding the use of Voxzogo in pregnant individuals, and there is insufficient understanding of the potential risks for major birth defects, miscarriage, or other adverse outcomes for both maternal and fetal health. 
• The presence of Voxzogo in human breast milk, its impact on nursing infants, and its influence on milk production remain undisclosed. 
• Safety and effectiveness data for Voxzogo in pediatric patients with achondroplasia below the age of 5 years have not been established. 
• The impact of renal impairment on the pharmacokinetics of Voxzogo has not been evaluated. 
• No dosage adjustment is needed for patients with eGFR ≥ 60 mL/min/1.73 m2. Voxzogo is not recommended for patients with eGFR < 60 mL/min/1.73 m2.

Storage and Handling

• Store the Voxzogo vial and prefilled diluent syringe in the refrigerator between 36°F to 46°F (2°C to 8°C). 
• Before mixing, you can also store Voxzogo at room temperature between 68°F to 77°F (20°C to 25°C) for a maximum of 90 days. 
• Note the start date of room temperature storage on the carton to monitor the 90 days. Once stored at room temperature, do not return Voxzogo to the refrigerator, and discard it if unused within the specified 90 days. 
• Ensure not to use Voxzogo beyond its expiration date, and refrain from freezing it. Store Voxzogo away from direct sunlight, and keep it, along with all medicines, out of the reach of children.