NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. The US FDA has approved eculizumab (Soliris, Alexion) for the treatment of anti-aquaporin-4 antibody positive (AQP4-IgG-positive) neuromyelitis optica spectrum disorder (NMOSD), which accounts for nearly three-quarters of the population with NMOSD. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. In June 2019, the FDA approved Soliris (eculizumab, Alexion Pharmaceuticals), the first therapy for the treatment of anti-aquaporin-4 (AQP4) auto­antibody-positive NMOSD. APPROVAL & LABELING We have completed our review of this application, as amended. Soliris is used to treat adults with anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). The drug is now approved for neuromyelitis optica spectrum disorder (NMOSD) in adult patients, who are anti-aquaporin-4 (AQP4) antibody positive. Despite the recent Soliris and Uplizna approvals, about 40% of patients with NMOSD remain untreated. Silver Spring, MD – The U.S. Food and Drug Administration yesterday approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. According to the National Institutes of Health, women are more often affected by NMOSD than men and African Americans are at greater risk of the disease than Caucasians. Prescribers must enroll in the REMS program. Soliris will nevertheless still face competition in the coming years from new challengers from the likes of Apellis, Roche, and Regeneron among others. The FDA granted this application Priority Review. Soliris FDA Approval History. Andrew McConaghie. var arr3= [ 'Alexion', 'Soliris', 'Rare diseases' ]; The most frequently reported adverse reactions reported by patients in the NMOSD clinical trial were: upper respiratory infection, common cold (nasopharyngitis), diarrhea, back pain, dizziness, influenza, joint pain (arthralgia), sore throat (pharyngitis) and contusion. Consequently, there is an unmet need for this orphan condition. WASHINGTON -- The first treatment for neuromyelitis optica spectrum disorder (NMOSD), a relapsing autoimmune inflammatory condition, received FDA approval Thursday. The FDA granted the approval of Soliris to Alexion Pharmaceuticals. For now there are no approved therapies for NMOSD in Europe. Use should be discontinued in patients who are being treated for serious meningococcal infections. The approval was granted under orphan drug designation, which will help extend the drug’s market exclusivity period. Home Working Medical Copy Writer & Senior Medical Copy Write... Editor & Senior Editor - Medical Communications - Home Based. It is not known if Soliris is safe and effective in children with NMOSD. FDA approves Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. 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. FDA Approved: Yes (First approved March 16, 2007) Brand name: Soliris Generic name: eculizumab Dosage form: Injection Company: Alexion Pharmaceuticals, Inc. Health care professionals should use caution when administering Soliris to patients with any other infection. Soliris also received FDA approval for the treatment of aHUS, gMG, and NMOSD. • The approval of Soliris for the new indication was based on a double -blind study in 143 patients with NMOSD who were anti-AQP4 antibody positive. For adult and pediatric patients with aHUS, and adult patients with gMG or NMOSD, supplemental dosing of Soliris is required in the setting of concomitant plasmapheresis or plasma exchange, or fresh frozen plasma infusion (PE/PI) (Table 2). “This approval changes the landscape of therapy for patients with NMOSD. “Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. NMOSD is an autoimmune disease of the central nervous system that mainly affects the optic nerves and spinal cord. The drug maker, Alexion Pharmaceuticals, received FDA approval for the same indication in June. Treatment for: Paroxysmal Nocturnal Hemoglobinuria, Hemolytic Uremic Syndrome, Myasthenia Gravis. With the approval of Soliris, there is now for the first time an FDA-approved treatment available to NMOSD patients to help reduce the risk of relapse.” The approval was built on data from the Phase III PREVENT trial. Individuals also can have attacks resulting in transverse myelitis, which often causes numbness, weakness, or paralysis of the arms and legs, along with loss of bladder and bowel control. SOLIRIS was approved for the treatment of NMOSD in adult patients who are anti-AQP4 antibody-positive by the U.S. Food and Drug Administration (FDA) in June 2019 and by the European Commission (EC) in August 2019. SOLIRIS received Orphan Drug Designation (ODD) for the treatment of NMOSD in the U.S., EU and Japan. The US regulator approved Soliris (eculizumab) yesterday to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. The drug must be dispensed with the FDA-approved patient Medication Guide that provides important information about the drug’s uses and risks. Having an approved therapy for this condition is the culmination of extensive work we have engaged in with drug companies to expedite the development and approval of safe and effective treatments for patients with NMOSD, and we remain committed to these efforts for other rare diseases.”. Soliris was first given the FDA nod in March 2007 for paroxysmal nocturnal hemoglobinuria. Soliris is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS). Neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease. Compared to treatment with placebo, the study showed that treatment with Soliris reduced the number of NMOSD relapses by 94 percent over the 48-week course of the trial. “This approval changes the landscape of therapy for patients with NMOSD. In clinical trials, Soliris reduced NMOSD relapses significantly, also reducing the need for hospitalizations. Alexion has gained a new FDA approved used for its rare disease blockbuster Soliris. This Prior Approval sBLA provides for the use of Soliris for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti­ aquaporin-4 (AQP4) antibody positive and for proposed modifications to the approved REMS. Soliris also reduced the need for hospitalizations and the need for treatment of acute attacks with corticosteroids and plasma exchange. A supplemental New Drug Application is currently under review by regulatory authorities in Japan. And while treatment has “come a long way," new treatment options remain “crucial,” she said. Soliris is a complement inhibitor indicated for: Account Manager - Creative Healthcare Communications - South Wes... SENIOR ACCOUNT MANAGER - MEDICAL COMMUNICATIONS. Benefit: Soliris will impact the medical benefit as administration is an intravenous medication. In the NMOSD clinical trial, no cases of meningococcal infection were observed. The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. A new approval in NMOSD will help Alexion's Soliris angle for $1 billion in sales by 2028, but getting first-line patients on board could be an issue. • Soliris is the first FDA-approved treatment for NMOSD. The FDA on Thursday approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. European Commission Approves SOLIRIS® (eculizumab) For the Treatment of Adults with Neuromyelitis Optica Spectrum Disorder (NMOSD) - SOLIRIS®(eculizumab) is the first and only approved medication for NMOSD in Europe-. The FDA approved the new indication on June 27, 2019. The U.S. Food and Drug Administration today approved Soliris (eculizumab) injection for intravenous use for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Sales Director, Healthcare communications agency, London/ SE UK, Account Executive - Medical Communications - Cheshire. Approximately 50% of patients with NMOSD have permanent visual impairment and paralysis caused by NMOSD attacks. Estimates vary, but NMOSD is thought to impact approximately 4,000 to 8,000 patients in the United States. “ We are thrilled to have partnered with industry to catalyze research and development of targeted therapies to treat NMOSD. The effectiveness of Soliris for the treatment of NMOSD was demonstrated in a clinical study of 143 patients with NMOSD who had antibodies against AQP4 (anti-AQP4 positive) who were randomized to receive either Soliris treatment or placebo. been conducted for NMOSD. 28th August 2019. by. var arr = [ 'alexion', 'soliris', 'rare_diseases' ]; Unlike Enspryng and Soliris, Uplizna showed an impact on the disability endpoint in pivotal testing. Eculizumab (Soliris®) is a humanized monoclonal antibody directed against the human complement component 5 (C5), inhibiting C5 enzymatic cleavage and thereby preventing the generation o f the First, the European Commission approved brand-name eculizumab (Soliris) for the treatment of adults with NMOSD who are anti-aquaporin-4 (AQP4) antibody positive. The US regulator approved Soliris (eculizumab) yesterday to treat neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. The market will be sorting out the respective strengths and weaknesses of the trio in NMOSD. In patients with NMOSD, the body's immune system mistakenly attacks healthy cells and proteins in the body, most often in the optic nerves and spinal cord. Article by Soliris is cleared for use in patients who are anti-aquaporin-4 (AQP4) antibody positive in the US, but Roche is hoping to get a … An official website of the United States government, Recalls, Market Withdrawals and Safety Alerts, FDA approves first treatment for neuromyelitis optica spectrum disorder, a rare autoimmune disease of the central nervous system, National Institute of Neurological Disorders and Stroke: Neuromyelitis Optica Information Page, FDA approved drugs: Questions and Answers. 1. The approval, which is the first of its kind, followed a 6-month priority review based on data from the PREVENT trial, which demonstrated safety and efficacy … The FDA approval of SOLIRIS is … Both are infused therapies. About Soliris Soliris ® (eculizumab) is approved by the FDA. The drug has already had its patent period extended in the US extended until 2027, giving Alexion significantly longer to ramp up sales of its successor treatment, Ultomiris, which gained FDA approval in December in PNH.Approval in NMOSD is expected later this year in Europe and in 2020 in Japan. “Soliris provides the first FDA-approved treatment for neuromyelitis optica spectrum disorder, a debilitating disease that profoundly impacts patients’ lives,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. Patients were randomized to receive Soliris or placebo. Soliris is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. document.write('
'); OPEN Health is a family of expert practices, working in partnership to drive positive change in healthcare communications & market... Communiqué Awards 2015: Winners in pictures, UK introduces Rare Diseases Framework to improve the lives of people with rare diseases, The five-step, rare disease-ready checklist, NICE backs first ‘ultra-orphan’ drug Soliris, Alexion's Soliris gets US approval for myasthenia gravis, Pharma Market Research Report - Predictions for 2016, Patient Partnerships: Challenges and Opportunities for Pharma, Learning about Rare Genetic Disorders on Undiagnosed Children’s Day. The FDA approval of SOLIRIS is … Soliris was approved by the Australian Therapeutic Goods Administration for the treatment of PNH in February 2009. NMOSD can be associated with antibodies that bind to a protein called aquaporin-4 (AQP4). Estimates vary, but NMOSD is thought to affect around 4,000 to 8,000 patients in the US. Patients should be monitored for early signs of meningococcal infections and evaluated immediately if infection is suspected. The site is secure. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. In it, patients with NMOSD who tested anti-AQP4 antibody positive received Soliris or placebo. var arr2 = [ 'Alexion', 'Soliris', 'Rare diseases' ]; Before sharing sensitive information, make sure you're on a federal government site. for (counter=0; counter