SOLIRIS® (eculizumab) Receives Approval in Japan for the Prevention of Relapse in Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD)
- SOLIRIS® (eculizumab) is the first and only approved medication for NMOSD in
- 98% of adult anti-aquaporin-4 (AQP4) antibody-positive patients treated with SOLIRIS were relapse free compared to 63% receiving placebo at 48 weeks -
“We are pleased that the Japanese health authorities have approved SOLIRIS as a new treatment for patients suffering from this complex and unpredictable disease,” said
NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses, also referred to as attacks. Each attack can result in stepwise accumulation of disability, including blindness and paralysis and sometimes premature death. NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years. Previously known as Devic’s Disease, NMOSD is often confused with other neurological illnesses such as multiple sclerosis (MS), which can lead to delays in diagnosis and treatment with medicines that can worsen disease progression.
“The understanding of NMOSD has rapidly evolved in recent years since complement activation by AQP4 antibodies was identified as an underlying cause of the disease,” said
The approval of SOLIRIS was based on comprehensive results from the Phase 3 randomized, double-blind placebo controlled PREVENT trial, which were published in
The safety profile of SOLIRIS was consistent with that seen for SOLIRIS in other clinical studies and real-world use in its three approved indications. The most common adverse events observed in the PREVENT study were upper respiratory tract infection (29 percent of patients in the SOLIRIS group vs. 13 percent in the placebo group), headache (23 vs. 23 percent), nasopharyngitis (21 vs. 19 percent) and nausea (17 vs. 26 percent). The serious adverse events that were reported for more than one patient in either group were pneumonia (three patients in the SOLIRIS group vs. one patient in the placebo group) and cellulitis, sepsis and urinary tract infection (two patients for each event in the SOLIRIS group vs. no patient in the placebo group). One patient receiving SOLIRIS and concomitant supportive IST died from a pulmonary empyema. The patient had an extensive history of pulmonary disease and was an active smoker. No cases of meningococcal infection were observed in the study.
SOLIRIS was approved for the treatment of NMOSD in adult patients who are anti-AQP4 antibody-positive by the
NMOSD is a rare and severe, autoimmune, inflammatory disorder that attacks the central nervous system (CNS), in which complement activation due to anti-aquaporin-4 (AQP4) antibodies plays a significant role in the disease process. Patients with NMOSD experience unpredictable attacks, also referred to as relapses, which can cause irreversible damage to the optic nerve and spinal cord and can lead to long-term disability. The most common symptoms of NMOSD are optic neuritis and transverse myelitis. Optic neuritis can cause visual problems including blindness; transverse myelitis can cause mobility problems including paralysis. The disease primarily affects women, often in the prime of their lives, with an average age of onset of 39 years. The prevalence of NMOSD may be more common and more severe in non-Caucasian populations worldwide.
Approximately three quarters (73%) of all patients with NMOSD have AQP4 auto-antibodies. In patients with anti-AQP4 antibody-positive NMOSD, the body’s own immune system can turn against itself to produce auto-antibodies against AQP4, a protein on certain cells in the optic nerve, brain and spinal cord that are critical for the survival of nerve cells. The binding of these anti-AQP4 auto-antibodies activates the complement cascade, another part of the immune system. Complement activation by anti-AQP4 auto-antibodies can cause destruction of vital cells in the CNS, leading to demyelination and to the death of neurons, predominantly in the spinal cord and optic nerve.
SOLIRIS® (eculizumab) is a first-in-class complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade, a part of the immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe rare and ultra-rare disorders. SOLIRIS, an intravenously administered therapy, is approved in the U.S., EU,
Important Safety Information
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab)
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). SOLIRIS is used to treat 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). SOLIRIS is used to treat adults with a disease called generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. SOLIRIS is used to treat 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
SOLIRIS is a medicine that affects the immune system. SOLIRIS can lower the ability of the immune system to fight infections. SOLIRIS increases the 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.
Meningococcal vaccines must be received at least two weeks before the first dose of SOLIRIS if one has not already had this vaccine. If one’s doctor decided that urgent treatment with SOLIRIS is needed, meningococcal vaccination should be administered as soon as possible. If one has not been vaccinated and SOLIRIS therapy must be initiated immediately, two weeks of antibiotics should also be administered with the vaccinations. If one had a meningococcal vaccine in the past, additional vaccination might be needed before starting SOLIRIS. Patients should ask their doctor if an additional meningococcal vaccination is needed. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call one’s doctor or get emergency medical care right away if any of these signs and symptoms of a meningococcal infection occur: 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, and eyes sensitive to light. One’s doctor will provide a Patient Safety Card about the risk of meningococcal infection. Carry the card at all times during treatment and for 3 months after the last SOLIRIS dose.
SOLIRIS is only available through a program called the SOLIRIS REMS.
SOLIRIS may also increase the risk of other types of serious infections. If one’s child is treated with SOLIRIS, make sure that the 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 the doctor about whether one is at risk for gonorrhea infection, about gonorrhea prevention, and regular testing. Certain fungal infections (Aspergillus) may also happen if one takes SOLIRIS and has a weak immune system or a low white blood cell count.
Do not receive SOLIRIS if one has a meningococcal infection, or has not been vaccinated against meningitis infection unless one’s doctor decides that urgent treatment with SOLIRIS is needed.
Before one receives SOLIRIS, tell the doctor about all of the medical conditions, including if one: has an infection or fever, is pregnant or plans to become pregnant, and is breastfeeding or plans to breastfeed. It is not known if SOLIRIS will harm an unborn baby or if SOLIRIS passes into the breast milk.
Tell the doctor about all the medicines one takes, 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 one: has all recommended vaccinations before starting SOLIRIS, receives 2 weeks of antibiotics if one immediately starts SOLIRIS, and stays up-to-date with all recommended vaccinations during treatment with SOLIRIS. Know the medications one takes and the vaccines one receives. Keep a list of them to show the doctor and pharmacist when one gets a new medicine.
If one has PNH, the doctor will need to monitor closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of the red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of the red blood cell count, drop in the platelet counts, confusion, kidney problems, blood clots, difficulty breathing, and chest pain. If one has aHUS, the doctor will need to monitor 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, and a drop in the platelet count. SOLIRIS can cause serious side effects including serious allergic reactions. Serious allergic reactions can happen during one’s SOLIRIS infusion. Tell the doctor or nurse right away if one gets any of these symptoms during the SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of the face, tongue, or throat, and feeling faint or pass out. If one has an allergic reaction to SOLIRIS, the doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS.
The most common side effects in people with PNH treated with SOLIRIS include: headache, pain or swelling of the nose or throat (nasopharyngitis), back pain, and 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 the nose or throat (nasopharyngitis), low red blood cell count (anemia), cough, swelling of legs or feet (peripheral edema), nausea, urinary tract infections, and 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 the 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), and bruising (contusion).
Please see the accompanying full Prescribing Information and Medication Guide for SOLIRIS, including BOXED WARNING regarding serious and life-threatening meningococcal infections.
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases as well as several early-stage therapies, including one for light chain (AL) amyloidosis and a second anti-FcRn therapy. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. Headquartered in
This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion, including statements related to: the potential benefits of SOLIRIS as a treatment and prevention of relapse in adult patients with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) and the potential impact of SOLIRIS on patients suffering from NMOSD; and that Soliris can help prevent future relapses. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those expected by these forward looking statements, including for example: the anticipated benefits of SOLIRIS for NMOSD patients may not be realized (and the results of the clinical trials may not be indicative of the results once approved for use in the
Megan Goulart, 857-338-8634
Senior Director, Corporate Communications
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