Soliris (eculizumab) Page Indication Date Approved Date Updated 2 Neuromyelitis Optica (Devic’s Syndrome) June 27, 2019 June 28, 2019 3 Myasthenia Gravis Oct 23, 2017 May 24, 2019 4 Atypical Hemolytic Uremic Syndrome Sept 23, 2011 May 24, 2019 5 Paroxysmal Nocturnal Hemoglobinuria March 16, 2007 May 24, 2019 1 Drug Information Regulatory Information Manufacturer: Alexion Pharmaceuticals Application Type: Supplemental BLA Original Anticipated Approval Drug Brand 6/28/2019 Soliris (PDUFA): Name: Regulatory Status: Approved Drug Generic Regulatory Status Date: 6/27/2019 eculizumab Name: Regulatory Status Reason: N/A Orphan Drug Designation: 6/24/2013 Drug Type: Monoclonal Antibody FDA Fast Track Designation: N/A Breakthrough Therapy Route of N/A Intravenous Designation Administration: Priority Review Designation: 2/22/2019 Indication Details Sales Forecast Indication Prevalence Prevalence Type Neuromyelitis U.S. 1-10 / 100,000 Optica (Devic’s Global 1-10 / 100,000 Syndrome) Source: GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 23, 2019. Comments Section On February 22, 2019, FDA accepted for review the supplemental BLA for Soliris (eculizumab) as a treatment for patients with neuromyelitis optica spectrum disorder (NMOSD) who have anti-aquaporin-4 (AQP4) auto antibodies. The FDA granted Priority Review and set a PDUFA date of June 28, 2019. UPDATE: On June 27, 2019, FDA approved Soliris (eculizumab) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Soliris is the first FDA-approved treatment for neuromyelitis optica spectrum disorder. Sources: “FDA Approves First Treatment for Neuromyelitis Optica Spectrum Disorder, a Rare Autoimmune Disease of the Central Nervous System.” U.S. Food & Drug Administration, 27 June 2019, www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-neuromyelitis-optica-spectrum-disorder-rare- autoimmune-disease-central. “FDA Grants Priority Review and Accepts SBLA of SOLIRIS® (Eculizumab) as a Treatment for Patients with Neuromyelitis Optica Spectrum Disorder (NMOSD).” Alexion Pharmaceuticals, Inc, 22 Feb. 2019, news.alexionpharma.com/press-release/product-news/fda-grants-priority-review-and-accepts-sbla-soliris-eculizumab- treatment-. Accessed: May 24, 2019. GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. “Neuromyelitis Optica Spectrum Disorder.” NORD - National Organization for Rare Disorders, rarediseases.org/rare-diseases/neuromyelitis-optica/. Accessed: May 24, 2019. SOLIRIS (eculizumab) [Package Insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2019. 2 Drug Information Regulatory Information Manufacturer: Alexion Pharmaceuticals Application Type: Supplemental BLA Original Anticipated Approval Drug Brand 10/23/2017 Soliris (PDUFA): Name: Regulatory Status: Approved Drug Generic Regulatory Status Date: 10/23/2017 eculizumab Name: Regulatory Status Reason: N/A Orphan Drug Designation: 6/12/2014 Drug Type: Monoclonal Antibody FDA Fast Track Designation: N/A Breakthrough Therapy Route of N/A Intravenous Designation Administration: Priority Review Designation: N/A Indication Details Sales Forecast Indication Prevalence Prevalence Type Myasthenia U.S. 53,229 Gravis Global 441,748 Source: GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 23, 2019. Comments Section On October 23, 2017, FDA approved Soliris (eculizumab) for the treatment of adult patients with generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody positive. Myasthenia Gravis Prevalence: affects approximately 14-40 per 100,000 individuals in the U.S.** Myasthenia Gravis affects about 20 per 100,000 people worldwide.* Sources: “Drugs@FDA: FDA Approved Drug Products - Soliris.” U.S. Food and Drug Administration, www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process. Accessed: May 24, 2019. GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. * “Myasthenia Gravis - Genetics Home Reference.” National Institutes of Health - U.S. National Library of Medicine, 14 May 2019, ghr.nlm.nih.gov/condition/myasthenia-gravis#statistics. Accessed: May 24, 2019. ** “Myasthenia Gravis - Rare Disease Database.” NORD - National Organization for Rare Disorders, rarediseases.org/rare-diseases/myasthenia-gravis/. Accessed: May 24, 2019. SOLIRIS (eculizumab) [Package Insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2018. 3 Drug Information Regulatory Information Manufacturer: Alexion Pharmaceuticals Application Type: Supplemental BLA Original Anticipated Approval Drug Brand N/A Soliris (PDUFA): Name: Regulatory Status: Approved Drug Generic Regulatory Status Date: 9/23/2011 eculizumab Name: Regulatory Status Reason: N/A Orphan Drug Designation: 4/29/2009 Drug Type: Monoclonal Antibody FDA Fast Track Designation: N/A Breakthrough Therapy Route of N/A Intravenous Designation Administration: Priority Review Designation: N/A Indication Details Sales Forecast Indication Prevalence Prevalence Type Atypical Hemolytic U.S. > 1,500 Uremic Syndrome (Nondiarrhea- Associated Global N/A Hemolytic Uremic Syndrome) Source: GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. Comments Section On September 23, 2011, FDA approved Soliris (eculizumab) for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. The annual incidence of aHUS is estimated to be 2 cases per million in the U.S.; the prevalence is estimated to be 3.3 per million among patients below the age of 18.** aHUS accounts for approximately 5-10% of all cases of hemolytic uremic syndrome.* Sources: * “Atypical Hemolytic Uremic Syndrome - Rare Disease Database.” NORD - National Organization for Rare Disorders, rarediseases.org/rare-diseases/atypical- hemolytic-uremic-syndrome/. Accessed: May 24, 2019. “Drugs@FDA: FDA Approved Drug Products - Soliris.” U.S. Food and Drug Administration, www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process. Accessed: May 24, 2019. GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. “Just How Many AHUS Patients Are There?” AHUS Alliance, www.ahusallianceaction.org/just-many-ahus-patients/. Accessed: May 24, 2019. SOLIRIS (eculizumab) [Package Insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2018. ** Yoshida Y, Kato H, Nangaku M. Atypical hemolytic uremic syndrome. Renal Replacement Therapy (2017) 3:5. Available at: https://rrtjournal.biomedcentral.com/articles/10.1186/s41100-016-0088-1. 4 Drug Information Regulatory Information Manufacturer: Alexion Pharmaceuticals Application Type: BLA Original Anticipated Approval Drug Brand N/A Soliris (PDUFA): Name: Regulatory Status: Approved Drug Generic Regulatory Status Date: 3/16/2007 eculizumab Name: Regulatory Status Reason: N/A Orphan Drug Designation: 8/20/2003 Drug Type: Monoclonal Antibody FDA Fast Track Designation: N/A Breakthrough Therapy Route of N/A Intravenous Designation Administration: Priority Review Designation: N/A Indication Details Sales Forecast Indication Prevalence Prevalence Type Paroxysmal U.S. 6,524 Nocturnal Hemoglobinuria Global 103,186 Source: GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. Comments Section On March 16, 2007, FDA approved Soliris (eculizumab) for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. The REMS for Soliris (eculizumab) was originally approved on June 4, 2010. Paroxysmal Nocturnal Hemoglobinuria (PNH) Prevalence: estimated to affect between 1 and 5 per million people in the U.S.* Sources: “Drugs@FDA: FDA Approved Drug Products - Soliris.” U.S. Food and Drug Administration, www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=BasicSearch.process. Accessed: May 24, 2019. GlobalData, Pharma Intelligence Center. https://pharma.globaldata.com, Accessed: May 24, 2019. * “Paroxysmal Nocturnal Hemoglobinuria - Genetics Home Reference.” National Institutes of Health - U.S. National Library of Medicine, 14 May 2019, ghr.nlm.nih.gov/condition/paroxysmal-nocturnal-hemoglobinuria#statistics. Accessed: May 24, 2019. SOLIRIS (eculizumab) [Package Insert]. Boston, MA: Alexion Pharmaceuticals, Inc.; 2018. 5 .