ULTOMIRIS® (ravulizumab-cwvz) AND aHUS

ULTOMIRIS is the first and only long-acting C5 inhibitor that provides immediate and complete inhibition that is approved for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).1

The Phase 3 study of ULTOMIRIS, administered intravenously every four to eight weeks in adults and pediatric patients one month of age and older with aHUS, met its primary endpoint of Complete TMA Response.

ABOUT AHUS

aHUS is a genetic, chronic, ultra-rare disease that can progressively damage vital organs, such as the kidneys. aHUS is caused by genetic abnormalities that result in chronic uncontrolled complement activation leading to complement-mediated thrombotic microangiopathy (TMA) — the formation of blood clots in small blood vessels throughout the body.2

aHUS affects both adults and children. Patients with aHUS face a lifelong risk of TMA, which may lead to sudden, catastrophic, and life-threatening damage to the kidney and other vital organs.2

Historically, 56% of the adults and 29% of the children required renal replacement therapy or died within one year of follow-up5. Moreover, 33 to 40 percent of patients died or progressed to end-stage renal disease with the first clinical manifestation of aHUS despite supportive care.3,4

TREATING PATIENTS WHO HAVE AHUS WITH ULTOMIRIS®

The safety and efficacy of ULTOMIRIS® for the treatment of aHUS to inhibit complement-mediated TMA has been demonstrated in a global, multicenter, single arm, Phase 3 study which evaluated ULTOMIRIS administered by intravenous infusion in 56 adults (≥ 18 years of age) who were naïve to complement inhibitor treatment prior to study entry. The study consisted of an up to seven-day screening period, a 26-week initial evaluation period and an extension period of up to 4.5 years, which is still ongoing. The safety and efficacy of ULTOMIRIS is also being assessed in complement inhibitor-naïve children and adolescents (aged 18 and under) with a documented diagnosis of aHUS in a 26-week, ongoing, multicenter, single-arm, Phase 3 study.

The most frequently observed adverse reactions reported in this study were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia.

QUICK LINKS

Download ULTOMIRIS® Prescribing Information (PDF).

Visit the ULTOMIRIS® website to learn more about aHUS and treatment with ULTOMIRIS®.

INDICATION & IMPORTANT SAFETY INFORMATION for
ULTOMIRIS® (ravulizumab-cwvz) FOR INTRAVENOUS USE [300 MG/30 ML VIAL]

INDICATION

What is ULTOMIRIS?

ULTOMIRIS is a prescription medicine called a monoclonal antibody. ULTOMIRIS is used to treat:

  • 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).

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system. ULTOMIRIS can lower the ability of your immune system to fight infections.

  • ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.
    1. You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you have not already had this vaccine.
    2. If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
    3. If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
    4. If you had a meningococcal vaccine in the past, you might need additional vaccination before starting ULTOMIRIS. Your doctor will decide if you need additional meningococcal vaccination.
    5. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection:
    • headache with nausea or vomiting
    • headache and fever
    • headache with a stiff neck or stiff back
    • fever
    • fever and a rash
    • confusion
    • muscle aches with flu-like symptoms
    • eyes sensitive to light

Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. Your risk of meningococcal infection may continue for several months after your last dose of ULTOMIRIS. It is important to show this card to any doctor or nurse who treats you. This will help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must:

  • enroll in the ULTOMIRIS REMS program
  • counsel you about the risk of meningococcal infection
  • give you information about the symptoms of meningococcal infection
  • give you a Patient Safety Card about your risk of meningococcal infection, as discussed above
  • make sure that you are vaccinated with a meningococcal vaccine

ULTOMIRIS may also increase the risk of other types of serious infections.

  • People who take ULTOMIRIS may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae.
  • Certain people may also have an increased risk of gonorrhea infection. Talk to your doctor to find out if you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.
  • Call your doctor right away if you have any new signs or symptoms of infection.

    Who should not receive ULTOMIRIS?

    Do not receive ULTOMIRIS if you:

    • have a meningococcal infection
    • Have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed. See “What is the most important information I should know about ULTOMIRIS.”

    Before you receive ULTOMIRIS, tell your doctor about all of your medical conditions, including if you:

    • have an infection or fever.
    • are pregnant or plan to become pregnant. It is not known if ULTOMIRIS will harm your unborn baby.
    • are breastfeeding or plan to breastfeed. It is not known if ULTOMIRIS passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

    Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and other medicines can affect each other causing side effects.
    Know the medicines you take and the vaccines you receive. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

    How should I receive ULTOMIRIS?

    • ULTOMIRIS is given through a vein by intravenous (I.V.) infusion usually over about 2 hours in adults and up to 4 hours in children.
    • If you are an adult with 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 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.
    • Your doctor will decide how long you need to take ULTOMIRIS for your aHUS.
    • If you are changing treatment from SOLIRIS to ULTOMIRIS, you should receive your starting dose of ULTOMIRIS 2 weeks after your last dose of SOLIRIS.
    • After each infusion, you should be monitored for at least 1 hour for infusion reactions. See “What are the possible side effects of ULTOMIRIS?”
    • If you have aHUS, your doctor will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS symptoms or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include:
      • confusion or loss of consciousness
      • seizures
      • chest pain (angina)
      • difficulty breathing
      • blood clots or stroke
    • If you miss an ULTOMIRIS infusion, call your doctor right away.

    What are the possible side effects of ULTOMIRIS?
    ULTOMIRIS can cause serious side effects including:

    • See “What is the most important information I should know about ULTOMIRIS?”
    • Infusion reactions. Infusion reactions may happen during your ULTOMIRIS infusion. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including:
      • chest pain
      • trouble breathing or shortness of breath
      • swelling of your face, tongue, or throat
      • feel faint or pass out

    Your doctor will treat your symptoms as needed.

    The most common side effects of ULTOMIRIS in people with aHUS are:

    • upper respiratory infections
    • diarrhea
    • nausea
    • vomiting
    • headache
    • high blood pressure
    • fever

    Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your doctor or pharmacist.

    Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

    Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.

    References

    1. ULTOMIRIS® [package insert]. Alexion Pharmaceuticals Inc; 10/2019.
    2. Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N Engl J Med. 2009;361:1676-87.
    3. Noris M, Caprioli J, Bresin E, et al. Relative Role of Genetic Complement Abnormalities in Sporadic and Familial aHUS and Their Impact on Clinical Phenotype. CJASN.2010;5:1844-1859.
    4. Caprioli J, Noris M, Brioschi S, et al. The impact of MCP, CFH, and IF mutations on clinical presentation, response to treatment, and outcome. Blood. 2006;108:1267-1269.
    5. Fremeaux, Bacchi, et al. Genetics and Outcome of Atypical HUS in Children and Adults. Clin J Am Soc Nephrol 2013;8:554–562

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