Voxelotor

What is Voxelotor?

• Voxelotor (OXBRYTA) is a hemoglobin S polymerization inhibitor used for the treatment of sickle cell disease.

What are the uses of this medicine?

• This medicine is used for the treatment of sickle cell disease in adults and children 12 years of age and older.

How does this medicine work?

• An orally bioavailable modulator and stabilizer of sickle cell hemoglobin (HbS), with potential use in the treatment of sickle cell disease (SCD). Upon administration, voxelotor targets and covalently binds to the N-terminal valine of the alpha chain of HbS.
• This stabilizes HbS, thereby improving oxygen binding affinity.
• The binding of voxelotor to HbS prevents HbS polymerization, reduces sickling, decreases red blood cell (RBC) damage and increases the half-life of RBCs.
• This improves blood flow and decreases hemolytic anemia.

Who Should Not Use this medicine ?

This medicine cannot be used in patients with:

• history of serious drug hypersensitivity reaction to voxelotor or excipients. Clinical manifestations may include generalized rash, urticaria, mild shortness of breath, mild facial swelling, and eosinophilia.

What drug interactions can this medicine cause?

Avoid co-administration of OXBRYTA with:

• Sensitive CYP3A4 Substrates
• Strong CYP3A4 Inhibitors or Fluconazole
• Strong or Moderate CYP3A4 Inducers

Is this medicine FDA approved?

• It was approved for use in the United States in 2019.

How should this medicine be used?

Recommended Dosage

• The recommended dosage of OXBRYTA is 1,500 mg taken orally once daily with or without food. If a dose is missed, continue dosing on the day following the missed dose.
• OXBRYTA may be given with or without hydroxyurea.

Recommended Dosage for Hepatic Impairment

• The recommended dosage of OXBRYTA in patients with severe hepatic impairment (Child Pugh C) is 1,000 mg taken once daily with or without food.

Administration

• Take OXBRYTA exactly as your healthcare provider tells you.
• Do not change your dose or stop taking OXBRYTA unless your healthcare provider tells you to.
• Take OXBRYTA 1 time each day. Swallow each OXBRYTA tablet whole. Do not cut, crush or chew the tablets.
• Your healthcare provider may change your dose if needed.
• Your healthcare provider may also prescribe hydroxyurea during treatment with OXBRYTA.
• Take OXBRYTA with or without food.
• If you forget to take a dose of OXBRYTA, skip that dose and return to your normal dosing schedule the next day.

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

This medicine is available in fallowing doasage form: As Tablets 500 mg

This medicine is available in fallowing brand namesː

• OXBRYTA

What side effects can this medication cause?

The most common side effects of this medicine include:

• headache
• diarrhea
• stomach (abdominal) pain
• nausea
• tiredness
• rash
• fever

OXBRYTA can cause serious side effects, including:

• Serious allergic reactions including:
• rash
• hives
• shortness of breath
• swelling of the face

What special precautions should I follow?

• Serious hypersensitivity reactions after administration of OXBRYTA have occurred. Observe for signs and symptoms and manage promptly.
• OXBRYTA administration may interfere with measurement of Hb subtypes (HbA, HbS, and HbF) by high-performance liquid chromatography (HPLC).Perform quantification of hemoglobin species when patient is not receiving OXBRYTA.

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?

• There are no available data on OXBRYTA 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 OXBRYTA for sickle cell disease have been established in pediatric patients aged 12 years and older.
• The safety and efficacy of OXBRYTA in pediatric patients below the age of 12 years have not been established.

What are the active and inactive ingredients in this medicine?

• Active Ingredient: voxelotor
• Inactive Ingredients: colloidal silicon dioxide, croscarmellose sodium, magnesium stearate, microcrystalline cellulose, and sodium lauryl sulfate. The film coating contains: polyethylene glycol 3350, polyvinyl alcohol, talc, titanium dioxide, and yellow iron oxide.

Who manufactures and distributes this medicine?

• Manufactured for: Global Blood Therapeutics, Inc. South San Francisco, CA 94080, USA.
• OXBRYTA is a registered trademark of Global Blood Therapeutics, Inc.

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

• Store OXBRYTA at or below 86°F (30°C).
• OXBRYTA comes in a child-resistant package.
• The bottle contains a desiccant to help keep your medicine dry (protect it from moisture) and polyester coil. Do not eat.
• Keep OXBRYTA and all medicines out of the reach of children.

• Dailymed label info on Voxelotor
• FDA Voxelotor