Ravulizumab

What is Ravulizumab?

• Ravulizumab (Ultomiris) is a complement inhibitor used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome.

What are the uses of this medicine?

This medicine is used to treat:

• adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
• adults and children 1 month of age and older with a disease called atypical hemolytic uremic syndrome (aHUS).
• Ultomiris is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

How does this medicine work?

• Ravulizumab-cwvz is a terminal complement inhibitor that specifically binds to the complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the terminal complement complex [C5b-9]) and preventing the generation of the terminal complement complex C5b9.
• Ultomiris inhibits terminal complement-mediated intravascular hemolysis in patients with PNH and complement-mediated thrombotic microangiopathy (TMA) in patients with aHUS.

Who Should Not Use this medicine ?

This medcine cannot be used in patients with:

• unresolved Neisseria meningitidis infection
• have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with Ultomiris is needed.

What drug interactions can this medicine cause?

• No clinically important drug interactions have been observed with Ultomiris.

Is this medicine FDA approved?

• It was approved for use in the United States in 2018.

How should this medicine be used?

• Vaccinate patients for meningococcal disease according to current ACIP guidelines to reduce the risk of serious infection.
• Provide 2 weeks of antibacterial drug prophylaxis to patients if Ultomiris must be initiated immediately and vaccines are administered less than 2 weeks before starting Ultomiris therapy.

Recommended Dosage

• The recommended dosing regimen in adult and pediatric patients, one month of age or older weighing 5 kg or greater, with PNH and aHUS consists of a loading dose followed by maintenance dosing, administered by intravenous infusion. The dosing is based on the patient's body weight, as shown belowː

Body Weight Range (kg)	Loading Dose (mg)	Maintenance Dose (mg) and Dosing Interval
5 to less than 10	600	300	Every 4 weeks
10 to less than 20	600	600	Every 4 weeks
20 to less than 30	900	2,100	Every 8 weeks
30 to less than 40	1,200	2,700	Every 8 weeks
40 to less than 60	2,400	3,000	Every 8 weeks
60 to less than 100	2,700	3,300	Every 8 weeks
100 or greater	3,000	3,600	Every 8 weeks

Administration

• Ultomiris is given through a vein by intravenous (I.V.) infusion

If you are an adult with PNH or aHUS, you will usually receive:

• a starting dose of Ultomiris as an infusion by your doctor, and then
• 2 weeks later, you will start to receive an infusion of Ultomiris every 8 weeks.

Children 1 month of age and older with PNH or aHUS will usually receive:

• a starting dose of Ultomiris as an infusion by your doctor, and then
• your doctor will decide how often your child will receive Ultomiris, either every 4 weeks or every 8 weeks, depending on their weight, starting 2 weeks after the starting dose.

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

This medicine is available in fallowing doasage form: As Injection:

• 300 mg/30 mL (10 mg/mL) in a single-dose vial .
• 300 mg/3 mL (100 mg/mL) in a single-dose vial.
• 1,100 mg/11 mL (100 mg/mL) in a single-dose vial.

This medicine is available in fallowing brand namesː

• Ultomiris

What side effects can this medication cause?

The most common side effects of Ultomiris in people treated for PNH are:

• upper respiratory tract infection
• headache

The most common side effects of Ultomiris in people treated for aHUS are:

• upper respiratory tract infection
• diarrhea
• nausea
• vomiting
• headache
• high blood pressure
• fever

Ultomiris can cause serious side effects including: Infusion-related reactions, including:

• chest pain
• trouble breathing or shortness of breath
• swelling of your face, tongue, or throat
• feel faint or pass out

What special precautions should I follow?

• Life-threatening meningococcal infections have occurred in patients treated with Ultomiris. Vaccinate for meningococcal disease according to the most current Advisory Committee on Immunization Practices (ACIP) recommendations for patients with complement deficiencies. Immunize patients without a history of meningococcal vaccination at least 2 weeks prior to receiving the first dose of Ultomiris.
• Due to the risk of meningococcal infections, Ultomiris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
• Ultomiris blocks terminal complement activation; therefore, patients may have increased susceptibility to encapsulated bacteria infections, especially infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Use caution when administering Ultomiris to patients with any other systemic infection.
• Administration of Ultomiris may result in infusion-related reactions. Monitor patients during infusion, interrupt for reactions, and institute appropriate supportive measures.

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 Ultomiris use in pregnant women to inform 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 Ultomiris for the treatment of PNH have been established in pediatric patients aged one month and older.
• The safety and effectiveness of Ultomiris for the treatment of aHUS have been established in pediatric patients aged one month and older.

What are the active and inactive ingredients in this medicine?

Active ingredient: ravulizumab-cwvz Inactive ingredients:

• Ultomiris 100 mg/mL: L-arginine, polysorbate 80 (vegetable origin), sodium phosphate dibasic, sodium phosphate monobasic, sucrose and Water for Injection
• Ultomiris 10 mg/mL: polysorbate 80 (vegetable origin), sodium chloride, sodium phosphate dibasic, sodium phosphate monobasic and Water for Injection

Who manufactures and distributes this medicine?

• Manufactured by Alexion Pharmaceuticals, Inc., 121 Seaport Boulevard, Boston, MA 02210 USA.

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

• Store Ultomiris vials refrigerated at 2°C - 8°C (36°F - 46°F) in the original carton to protect from light.
• Do not freeze.
• Do not shake.

• Dailymed label info on Ravulizumab
• FDA Ravulizumab

Ravulizumab Resources
Latest articles Most recent articles Ongoing trials	Wikipedia

Ravulizumab Resources
Latest articles Most recent articles Ongoing trials	Wikipedia