MEDICINES

SOLIRIS® (eculizumab)

About SOLIRIS

SOLIRIS is a first-in-class terminal complement inhibitor discovered, developed, and commercialized by Alexion. SOLIRIS works by selectively inhibiting activation of specific proteins in the complement system (C5a and C5b), which play a role in the pathophysiology of multiple rare diseases.

Soliris vial

SOLIRIS is indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, the treatment of adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive, and neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). It is not known if SOLIRIS is safe and effective in children with PNH, or gMG, or NMOSD.

SOLIRIS has earned some of the pharmaceutical industry’s highest honors for innovation, including the 2008 Prix Galien USA Award for Best Biotechnology Product and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases.

NMOSD

SOLIRIS and NMOSD

SOLIRIS is the first and only complement inhibitor approved by the FDA for the treatment of adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD).1

SOLIRIS is the first and only FDA-approved drug for the treatment of NMOSD in adult patients who are anti-AQP4 antibody positive. The safety and efficacy of SOLIRIS were established in a phase 3, randomized, double-blind, placebo-controlled, multicenter, time-to-event trial in adults with anti-AQP4 antibody-positive NMOSD (PREVENT, N=143).

The primary endpoint of the PREVENT trial was time to first adjudicated on-trial relapse, which was significantly longer in SOLIRIS-treated patients (n=96) compared to placebo-treated patients (n=47) (relative risk reduction, 94%; hazard ratio, 0.058; P<0.0001).

The most frequently reported adverse reactions in the PREVENT trial (≥10%) were: upper respiratory infection, nasopharyngitis, diarrhea, back pain, dizziness, influenza, arthralgia, pharyngitis, and contusion.

Read the multimedia news release

gMG

SOLIRIS and gMG

SOLIRIS is the first and only complement inhibitor approved for adults with anti-acetylcholine receptor antibody-positive (AChR+) generalized Myasthenia Gravis (gMG), a chronic and debilitating neuromuscular disorder.1,2

SOLIRIS is the first and only complement inhibitor approved for the treatment of adult patients with gMG who are AChR+. The safety and efficacy of SOLIRIS for the treatment of gMG were established in a 26-week, Phase 3, randomized, double-blind, placebo-controlled, multicenter clinical study (REGAIN, N=125).1,2

The primary efficacy endpoint was a comparison of the change from baseline between SOLIRIS (n=62) and placebo (n=63) in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26.2 See detailed results of the REGAIN Study and Interim Analysis from Phase 3 Open-Label Extension Study.

The most frequently reported adverse reaction in the gMG placebo-controlled trial (≥10%) is musculoskeletal pain.1

aHUS

SOLIRIS and aHUS

SOLIRIS was the first therapy approved for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.1 SOLIRIS is approved for the treatment of patients with aHUS in more than 40 countries, including the United States, European Union, and Japan.

The safety and efficacy of SOLIRIS for the treatment of aHUS has been demonstrated in 4 multinational, prospective studies (N=100). SOLIRIS treatment resulted in improvement in hematologic markers of complement-mediated TMA, including platelet counts and lactate dehydrogenase (LDH) levels.1,5-7 Patients treated with SOLIRIS also had improvements in renal function as measured by estimated glomerular filtration rate (eGFR).1,3-5

The most frequently reported adverse reactions in aHUS single-arm prospective trials (≥20%) are headache, diarrhea, hypertension, upper respiratory infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough, peripheral edema, nausea, urinary tract infections, and pyrexia.1

PNH

SOLIRIS and PNH

SOLIRIS was the first therapy approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis.1 SOLIRIS is approved for the treatment of patients with PNH in nearly 50 countries worldwide, including the United States, European Union, and Japan.

The safety and efficacy of SOLIRIS in patients with PNH with hemolysis were assessed in the randomized, double-blind, multicenter, placebo-controlled, 26-week TRIUMPH Study, which comprised 87 transfusion-dependent patients with PNH receiving SOLIRIS (n=43) or placebo (n=44).6 Patients were also treated in a single-arm, multicenter study comprising 97 patients with PNH treated with SOLIRIS over 52 weeks (SHEPHERD Study).7 A long-term extension study included 187 patients with PNH initially enrolled in one of 3 parent trials (N=195) who continued to receive SOLIRIS for a range of 10 to 54 months.1,8

Data from the trials demonstrated that patients treated with SOLIRIS experienced significantly reduced hemolysis, as measured by lactate dehydrogenase (LDH) levels, leading to an improvement in symptoms and a reduction of thrombotic events, a major health problem associated with the disease. Approximately half the patients received concomitant anticoagulant therapy. The effect of anticoagulant withdrawal during SOLIRIS treatment has not been studied.1,9

The most frequently reported adverse reactions in the PNH randomized trial (≥10% overall and greater than placebo) are headache, nasopharyngitis, back pain, and nausea.1

Learn more about treatment with SOLIRIS

VISIT THE SOLIRIS WEBSITE
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab)
INDICATIONS

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

  • patients with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH)
  • adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS)
    • SOLIRIS is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS)
  • adults with a disease called generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive
  • adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive

It is not known if SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.

IMPORTANT SAFETY INFORMATION

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

  • SOLIRIS 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 SOLIRIS if you have not already had this vaccine.
  2. If your doctor decided that urgent treatment with SOLIRIS is needed, you should receive meningococcal vaccination as soon as possible.
  3. If you have not been vaccinated and SOLIRIS therapy must be initiated immediately, you should also receive two weeks of antibiotics with your vaccinations.
  4. If you had a meningococcal vaccine in the past, you might need additional vaccination before starting SOLIRIS. 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 3 months after your last SOLIRIS dose. Your risk of meningococcal infection may continue for several weeks after your last dose of SOLIRIS. It is important to show this card to any doctor or nurse who treats you. This will help them diagnose and treat you quickly.

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

  • enroll in the SOLIRIS 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 the 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.

SOLIRIS may also increase the risk of other types of serious infections. If your child is treated with SOLIRIS, make sure that your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Certain people may be at risk of serious infections with gonorrhea. Talk to your doctor about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing. Certain fungal infections (Aspergillus) may also happen if you take SOLIRIS and have a weak immune system or a low white blood cell count.

Who should not receive SOLIRIS?
Do not receive SOLIRIS if you:

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

Before you receive SOLIRIS, 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 SOLIRIS will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if SOLIRIS passes into your breast milk.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. SOLIRIS and other medicines can affect each other causing side effects.

It is important that you:

  • have all recommended vaccinations before you start SOLIRIS
  • receive 2 weeks of antibiotics if you immediately start SOLIRIS
  • stay up-to-date with all recommended vaccinations during treatment with SOLIRIS

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.

Monitoring Disease After Stopping SOLIRIS

  • If you have PNH, your doctor will need to monitor you closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS 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 the number of your red blood cell count
    • drop in your platelet count
    • confusion
    • kidney problems
    • blood clots
    • difficulty breathing
    • chest pain

  • If you have aHUS, your doctor will need to monitor you closely during and for at least 12 weeks after stopping treatment for signs of worsening aHUS symptoms or problems related to abnormal clotting (thrombotic microangiopathy). Symptoms or problems that can happen with abnormal clotting may include:
    • stroke
    • confusion
    • seizure
    • chest pain (angina)
    • difficulty breathing
    • kidney problems
    • swellings in arms or legs
    • a drop in your platelet count

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

  • See “What is the most important information I should know about SOLIRIS?”
  • Serious allergic reactions. Serious allergic reactions can happen during your SOLIRIS infusion. Tell your doctor or nurse right away if you get any of these symptoms during your SOLIRIS infusion:
    • chest pain
    • trouble breathing or shortness of breath
    • swelling of your face, tongue, or throat
    • feel faint or pass out

If you have an allergic reaction to SOLIRIS, your doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS. See “How will I receive SOLIRIS?” in the Medication Guide.

The most common side effects in people with PNH treated with SOLIRIS include:

  • headache
  • pain or swelling of your nose or throat (nasopharyngitis)
  • back pain
  • nausea

The most common side effects in people with aHUS treated with SOLIRIS include:

  • headache
  • diarrhea
  • high blood pressure (hypertension)
  • common cold (upper respiratory infection)
  • stomach-area (abdominal) pain
  • vomiting
  • pain or swelling of your nose or throat (nasopharyngitis)
  • low red blood cell count (anemia)
  • cough
  • swelling of legs or feet (peripheral edema)
  • nausea
  • urinary tract infections
  • fever

The most common side effects in people with gMG treated with SOLIRIS include:

  • muscle and joint (musculoskeletal) pain

The most common side effects in people with NMOSD treated with SOLIRIS include:

  • common cold (upper respiratory infection)
  • pain or swelling of your nose or throat (nasopharyngitis)
  • diarrhea
  • back pain
  • dizziness
  • flu like symptoms (influenza) including fever, headache, tiredness, cough, sore throat, and body aches
  • joint pain (arthralgia)
  • throat irritation (pharyngitis)
  • bruising (contusion)

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 SOLIRIS. For more information, ask your doctor or pharmacist.
Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch, or call 1-800-FDA-1088.

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

References:

  1. Soliris [package insert]. Boston, MA: Alexion Pharmaceuticals, Inc; 2019.
  2. Howard JF Jr, Utsugisawa K, Benatar M, et al; for the REGAIN Study Group. Safety and efficacy of eculizumab in anti-acetycholine receptor antibody-positive refractory generalized Myasthenia Gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017;16(12):976-986.
  3. Fakhouri F, Hourmant M, Campistol J, et al. Terminal complement inhibitor eculizumab in adult patients with atypical hemolytic uremic syndrome: a single-arm, open-label trial. Am J Kidney Dis. 2016;68(1):84-93.
  4. Licht C, Greenbaum LA, Muus P, et al. Efficacy and safety of eculizumab in atypical hemolytic uremic syndrome from 2-year extensions of phase 2 studies. Kidney Int. 2015;87(5):1061-1073.
  5. Greenbaum LA, Fila M, Ardissiono G, et al. Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome. Kidney Int. 2016;90(3):701-711.
  6. Hillmen P, Young N, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355(12):1233-1243.
  7. Brodsky R. Advances in the diagnosis and therapy of paroxysmal nocturnal hemoglobinuria. Blood. 2008;22(2):65-74.
  8. Hillmen P, Muus P, Röth A, Elebute MO, Risitano AM, Schrezenmeier H. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013;162(1):62-73.
  9. Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123-4128.

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Chelsey, living with NMOSD, sitting on a couch
I don’t define myself as having a rare disease. It’s part of my life but it’s certainly not who I am.”
CHELSEY LIVING WITH NMOSD