Alexion Receives Positive CHMP Opinion for SOLIRIS® (eculizumab) for the Treatment of NMOSD - BioSpace
Alexion Receives Positive CHMP Opinion for SOLIRIS® (eculizumab) for the Treatment of NMOSD - BioSpace |
| Posted: 26 Jul 2019 08:30 AM PDT NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses, also referred to as attacks. Each attack results 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. "Each and every NMOSD attack has the potential to result in serious, irreversible consequences like blindness or losing the ability to walk," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "This is why attack, or relapse, prevention is the primary treatment goal for people living with NMOSD. There are currently no treatments approved in Europe for NMOSD; this positive opinion brings us one step closer to being able to offer SOLIRIS as the first-approved therapy for this very severe condition." The positive opinion is based on comprehensive results from the Phase 3 randomized, double-blind placebo controlled PREVENT trial, which were published in The New England Journal of Medicine and a long-term extension study (ECU-NMO-302), which is still underway. In the PREVENT study, patients with NMOSD who were anti-AQP4 antibody positive were treated with SOLIRIS (n=96) or placebo (n=47). The study met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse. At 48 weeks, 98 percent of patients treated with SOLIRIS were relapse free compared to 63 percent of patients receiving placebo. Of the patients treated solely with SOLIRIS, without receiving other immunosuppressive therapies, 100 percent were relapse free at 48 weeks compared to 61 percent in the placebo group. Sustained effects were observed through 144 weeks of treatment. 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 infectious pleural effusion. The patient had an extensive history of pulmonary disease and was an active smoker. No cases of meningococcal infection were observed in the study. The U.S. Food and Drug Administration (FDA) approved SOLIRIS (eculizumab) for the treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive on June 27, 2019. A supplemental New Drug Application currently is under review by regulatory authorities in Japan. SOLIRIS received Orphan Drug Designation (ODD) for the treatment of NMOSD in the U.S., EU and Japan. About NMOSD 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. 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. 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 eyes, 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. About SOLIRIS INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab) INDICATIONS IMPORTANT SAFETY INFORMATION 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. About Alexion For patient or advocacy inquiries please contact patientadvocacy@alexion.com. Forward-Looking Statement 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 final European Commission decision on the potential approval of SOLIRIS as a treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive with a relapsing course of the disease is anticipated in September 2019; SOLIRIS has the potential to be the first and only approved therapy for NMOSD in Europe for NMOSD; the Company's goal is to bringing SOLIRIS to patients with NMOSD in the EU; SOLIRIS can provide benefits for patients with NMOSD; and the anticipated timing of the review and decision of regulatory agencies with respect to the potential approval of SOLIRIS as a treatment for NMOSD. 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 European Commission may not approve SOLIRIS as a treatment for NMOSD (regardless of the opinion of the CHMP) or may be delayed in providing its approval beyond the anticipated approval date (and such delay may be significant); 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 European Union); results of clinical trials may not be sufficient to satisfy the European Commission or any other regulatory authority in order to approve SOLIRIS as a treatment for NMOSD (or they may request additional trials or additional information); results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations once the product is approved for use by regulatory agencies); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); our dependence on sales from our principal product (Soliris); future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by the European Commission and other regulatory agencies regarding products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions or grow the product pipeline through acquisitions (including due to failure to obtain antitrust approvals); the possibility that current rates of adoption of our products are not sustained; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to Ultomiris brought by third parties and inter partes review petitions submitted by third parties); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; the possibility that expected tax benefits will not be realized; potential declines in sovereign credit ratings or sovereign defaults in countries where we sell our products; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; uncertainties surrounding legal proceedings, company investigations and government investigations, including investigations of Alexion by the U.S. Securities and Exchange Commission (SEC) and U.S. Department of Justice; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other indications we are pursuing are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisition of Syntimmune and other companies and co-development efforts; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the quarter ended June 30, 2019 and in our other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law. View source version on businesswire.com: https://www.businesswire.com/news/home/20190726005239/en/ |
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