Vosoritide

What is Vosoritide?

• Vosoritide (voxzogo) is a C type natriuretic peptide (CNP) analog used for the treatment of achondroplasia.

Vosoritid

What are the uses of this medicine?

• Vosoritide (voxzogo) is a prescription medicine used to increase linear growth in children with achondroplasia who are 5 years of age and older with open bone growth plates (epiphyses).

How does this medicine work?

Vosoritide mechanism

• Achondroplasia is a genetic condition that causes severely short stature and disproportionate growth.
• The average height of an adult with achondroplasia is approximately four feet.
• People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth.
• In patients with achondroplasia, endochondral bone growth is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 (FGFR3).
• Binding of vosoritide to natriuretic peptide receptor-B (NPR-B) antagonizes FGFR3 downstream signaling by inhibiting the extracellular signal-regulated kinases 1 and 2 (ERK1/2) in the mitogen-activated protein kinase (MAPK) pathway at the level of rapidly accelerating fibrosarcoma serine/threonine protein kinase (RAF-1).
• As a result, vosoritide, like CNP, acts as a positive regulator of endochondral bone growth as it promotes chondrocyte proliferation and differentiation.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No drug-drug interaction studies were conducted.

Is this medicine FDA approved?

• Vosoritide was approved for medical use in the European Union in August 2021, and in the United States in November 2021.

How should this medicine be used?

• Have adequate food intake prior to voxzogo administration.
• Drink approximately 240-300 mL of fluid in the hour prior to voxzogo administration.

Recommended dosage:

• The recommended dosage of voxzogo is based on the patient's actual body weight.
• Monitor growth and adjust dosage according to body weight.
• Permanently discontinue upon closure of epiphyses.

Administration:

• voxzogo is given as an injection under the skin (subcutaneous or SC). Inject voxzogo 1 time every day, at about the same time each day.
• If your child's healthcare provider decides a caregiver can give the injections of voxzogo at home, your child's caregiver should receive training on the right way to prepare and inject voxzogo. Do not try to inject voxzogo until you have been shown the right way by your child's healthcare provider or nurse.
• Your child's healthcare provider will tell you how often you should give voxzogo. If your child misses a dose of voxzogo, it can be given within 12 hours of the scheduled time of injection. If more than 12 hours have passed, do not give the missed dose. Give the next daily dose according to your child's usual schedule.
• Your child should eat a meal and drink about 8 to 10 ounces of fluid within 1 hour before injection.
• In case you are not sure when to inject voxzogo, call your child's healthcare provider or pharmacist. Do not give voxzogo more often than as directed by your child's healthcare provider.
• Your child's dose of voxzogo depends on his or her body weight. Your child's healthcare provider will tell you which strength of voxzogo to use and how much to give your child.
• Your child's healthcare provider will monitor your child's growth and instruct you on when your child should stop voxzogo if they determine that your child is no longer able to grow. Stop giving voxzogo to your child if instructed by your child's healthcare provider.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As injection: 0.4 mg, 0.56 mg, or 1.2 mg lyophilized powder in a single-dose vial for reconstitution.

This medicine is available in fallowing brand namesː

• voxzogo

What side effects can this medication cause?

The most common side effects of this medicine include:

• injection site reactions (redness, itching, swelling, bruising, rash, hives, pain)
• vomiting
• joint pain
• decreased blood pressure
• stomach ache

voxzogo may cause serious side effects, including:

• risk of low blood pressure

What special precautions should I follow?

• Transient decreases in blood pressure were observed in clinical studies of voxzogo. To reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, fatigue and/or nausea), instruct patients to be well hydrated and have adequate food intake prior to administration of voxzogo.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• It is not known if voxzogo will harm your child's unborn baby.
• There are no available data on vosoritide use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.

Can this medicine be used in children?

• The safety and effectiveness of voxzogo have been established in pediatric patients aged 5 years and older for the improvement in linear growth in patients with achondroplasia.
• Safety and effectiveness of voxzogo in pediatric patients with achondroplasia below the age of 5 years have not been established.

What are the active and inactive ingredients in this medicine?

• Active ingredient: vosoritide
• Inactive ingredients: trehalose dihydrate, mannitol, sodium citrate dihydrate, methionine, citric acid monohydrate, and polysorbate 80
• BioMarin Pharmaceutical Inc.
• Novato, CA

Who manufactures and distributes this medicine?

Manufactured for:

• BioMarin Pharmaceutical Inc.
• 105 Digital Drive, Novato, CA

What should I know about storage and disposal of this medication?

• Store the voxzogo vial and prefilled diluent syringe in the refrigerator between 36°F to 46°F (2°C to 8°C).
• You may store voxzogo (before mixing) at room temperature between 68°F to 77°F (20°C to 25°C) for 90 days.
• Record the date you started storing voxzogo at room temperature on the carton to keep track of the 90 days.
• Do not return voxzogo to the refrigerator after it has been stored at room temperature.
• Throw voxzogo away if unused within 90 days of storing at room temperature.
• Do not use voxzogo past the expiration date.
• Do not freeze voxzogo.
• Store voxzogo out of direct sunlight.

• Dailymed label info on Vosoritide
• FDA Vosoritide

Vosoritide Resources
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