MEDICINES

ULTOMIRIS® (ravulizumab‑cwvz)

About ULTOMIRIS

ULTOMIRIS is the first and only long-acting C5 inhibitor administered every 8 weeks in adults. In maintenance dosing, ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. ULTOMIRIS is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of adults and pediatric patients 1 month of age and older with atypical hemolytic uremic syndrome (aHUS).

Ultomiris vial

ULTOMIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the United States and Japan.

ULTOMIRIS is approved in the United States and in Japan as a treatment for adults with PNH. ULTOMIRIS also is approved in the United States as a treatment for adults and pediatric patients 1 month of age and older with aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA). The medication is approved in the European Union for the treatment of adult patients with PNH with hemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with SOLIRIS® (eculizumab) for at least the past 6 months. The European Commission also approved ULTOMIRIS for the treatment of adults and children with a body weight of 10 kg or above with atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor-treatment naive or have received eculizumab for at least three months and have evidence of response to eculizumab.

In the United States, ULTOMIRIS is available in two formulations. ULTOMIRIS 100 mg/mL is an advanced formulation of ULTOMIRIS 10 mg/mL that provides a quicker infusion time for PNH and aHUS patients. ULTOMIRIS 100 mg/mL has the same mechanism of action as ULTOMIRIS 10 mg/mL, with consistent safety and efficacy. Both formulations are administered via intravenous infusion.

aHUS

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

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.

PNH

ULTOMIRIS and PNH

ULTOMIRIS is a treatment option for adult patients living with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS is the first and only long-acting C5 inhibitor that provides immediate and complete inhibition for 8 weeks.

In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH2 and patients with PNH who had been stable on SOLIRIS,3 intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all 11 endpoints.

Learn more about treatment with ULTOMIRIS

VISIT THE ULTOMIRIS WEBSITE
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab‑cwvz)
INDICATIONS

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

  • adults 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).

It is not known if ULTOMIRIS is safe and effective in children with PNH.

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 and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilis influenzae type b (Hib) if treated with ULTOMIRIS. 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 breast feed 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 medications 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 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 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 allergic reactions. See “What are the possible side effects of ULTOMIRIS?”
  • If you have PNH and you stop receiving ULTOMIRIS, your doctor will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include:
    • drop in your red blood cell count
    • tiredness
    • blood in your urine
    • stomach-area (abdomen) pain
    • shortness of breath
    • blood clots
    • trouble swallowing
    • erectile dysfunction (ED) in males
  • 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 infusion-related reactions.
Symptoms of an infusion-related 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, and feel faint or pass out. 

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory infection and headache.

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]. Boston, MA: Alexion Pharmaceuticals Inc; 2019.
  2. Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133(6):530-539.
  3. Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540-549.

US/ALL-A/0043

Jason, diagnosed with PNH
It’s important to me to remain positive. It’s a scary journey and there are unknowns. But from my perspective, I still have my life to live.”
JASON LIVING WITH PNH