– At 48 weeks, 98% of patients treated with SOLIRIS were relapse free compared to 63% of patients receiving placebo –
– SOLIRIS is the first and only FDA approved treatment for this rare, severe, condition that attacks the central nervous system without warning –
BOSTON, MA, USA I June 27, 2019 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that 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.1 Approximately three quarters (73%) of all patients with NMOSD test positive for anti-AQP4 auto-antibodies.2 The FDA approved SOLIRIS following an expedited six-month priority review. NMOSD is a rare, severe autoimmune disease that attacks the central nervous system without warning. These attacks, also referred to as relapses, can cause progressive and irreversible damage to the brain, optic nerve and spinal cord, which may lead to long-term disability.3–6 Complement activation due to anti-AQP4 antibodies is one of the primary underlying causes of the destruction in these patients.3,7 In the PREVENT trial, SOLIRIS, a first-in-class complement inhibitor, demonstrated safety and efficacy and met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse.1,8
“NMOSD is a serious disease with devastating consequences,” said Michael Levy, M.D., Ph.D., a consultant to the company and Associate Professor of Neurology at Massachusetts General Hospital in Boston. “Each attack can result in potentially irreversible consequences—causing blindness or losing the ability to walk—so preventing relapse is the primary goal of treatment. 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.”
NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years.3 Race is also a significant risk factor for disability and mortality in NMOSD.9–11 In the U.S., African Americans are over-represented among patients diagnosed with NMOSD and more likely to suffer more frequent and more severe attacks.9–11 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.3,12,13
“Today’s approval represents an important milestone for the NMOSD community,” said Victoria Jackson, co-founder of the Guthy-Jackson Charitable Foundation (GJCF), a non-profit organization dedicated to funding research and raising awareness about NMOSD. “We are thrilled to have partnered with industry to catalyze research and development of targeted therapies to treat NMOSD. The FDA approval of SOLIRIS is the beginning of a new era for these NMOSD patients as we continue on our mission to cure this life-threatening disease.”
This approval is based on comprehensive results from the Phase 3 randomized, double-blind placebo controlled PREVENT trial, which were recently published in The New England Journal of Medicine. In the study, patients with NMOSD who were anti-AQP4 antibody positive were treated with SOLIRIS (n=96) or placebo (n=47). At 48 weeks, 98 percent of patients treated with SOLIRIS were relapse free compared to 63 percent of patients receiving placebo. This effect was observed through 144 weeks of treatment, with 96 percent of patients treated with SOLIRIS relapse free compared to 45 percent of patients in the placebo arm. SOLIRIS-treated patients experienced similar improvement in time to first adjudicated on-trial relapse with or without concomitant treatment. Of the patients treated solely with SOLIRIS, without receiving other immunosuppressive therapies (IST), (n=21), 100 percent were relapse free at 144 weeks compared to 20 percent in the placebo group (n=13).8
The safety profile of SOLIRIS was consistent with that seen for SOLIRIS in other clinical studies. 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), nasopharyngitis (21 vs. 19 percent), diarrhea (16 vs. 15 percent), back pain (15 vs. 13 percent) and dizziness (15 vs. 13 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.
“In NMOSD, disease progression is driven by attacks, and so preventing future relapses is the primary goal of treatment. Today, for the first time, adults living with anti-AQP4 antibody-positive NMOSD have an approved treatment to reduce the risk of an unpredictable relapse – or attack,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion.
The European Medicines Agency (EMA) and the Japanese Ministry of Health, Labour and Welfare (MHLW) are reviewing Alexion’s applications to add the treatment of NMOSD to the marketing authorizations for SOLIRIS in the European Union (EU) and Japan, respectively. SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with NMOSD in the U.S., EU and Japan.
About NMOSD
NMOSD is a rare and devastating 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.3,7 Patients with NMOSD experience unpredictable attacks, also referred to as relapses, which can cause irreversible damage to the brain and spinal column— and can lead to long-term disability.3–6 The most common symptoms of NMOSD are optic neuritis and transverse myelitis. Transverse myelitis can cause mobility problems including paralysis; optic neuritis can cause visual problems including blindness.14,15
Race and gender are risk factors for NMOSD.9–11 The prevalence of NMOSD may be more common and more severe in non-Caucasian populations worldwide.9–11 People of African heritage, including African Americans, are disproportionately affected compared to the general NMOSD population.9,16 The disease primarily affects women, often in the prime of their lives, with an average age of onset of 39 years.3
Approximately three quarters (73%) of all patients with NMOSD have AQP4 auto-antibodies.2 Anti-AQP4 auto-antibody testing is part of the diagnostic criteria for NMOSD.17 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. 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.13,18–20
About SOLIRIS® (eculizumab)
SOLIRIS 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. Activation of the complement cascade, plays a role in severe rare and ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG), and anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). SOLIRIS is approved in the U.S., EU, Japan and other countries as a treatment for adult patients with PNH and for adults and children with aHUS. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). In the U.S., SOLIRIS is also approved for the treatment of adult patients with generalized MG (gMG) who are anti-AchR antibody-positive, in the EU as the first and only approved treatment of refractory gMG in adults who are anti-AchR antibody-positive and in Japan for the treatment of patients with gMG who are AchR antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin (IVIG) therapy or plasmapheresis (PLEX).
SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, Japan and many other countries, for the treatment of patients with aHUS in the U.S., EU and many other countries, for the treatment of patients with gMG in the U.S. for the treatment of patients with refractory gMG in EU and Japan, and for the treatment of NMOSD in the U.S., EU and Japan. Alexion and SOLIRIS have received some of the pharmaceutical industry’s highest honors for the medical innovation in complement inhibition: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
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). 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.
About Alexion
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), as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and anti-AQP4 antibody-positive 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 and metabolic disorders. Alexion has been named to the Forbes’ list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.
References
1 SOLIRIS® Product Information.
2 Hamid SHM, Whittam D, Mutch K, et al. What proportion of AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG positive? A cross sectional study of 132 patients. J Neurol. 2017;264(10):2088-2094.
3 Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805-815.
4 Wingerchuk DM. Diagnosis and Treatment of Neuromyelitis Optica. Neurologist. 2007;13(1):2-11.
5 Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options Neurol. 2008;10(1):55-66.
6 Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan. Brain. 2012;135(6):1834-1849.
7 Papadopoulos MC, Verkman AS. Aquaporin water channels in the nervous system. Nat Rev Neurosci. 2013;14(4):265.
8 Pittock SJ, Berthele A, Fujihara K, et al. Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019.
9 Flanagan EP, Cabre P, Weinshenker BG, et al. Epidemiology of aquaporin‐4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775-783.
10 Kim S-H, Mealy MA, Levy M, et al. Racial differences in neuromyelitis optica spectrum disorder. Neurology. 2018;91(22):e2089-e2099.
11 Mealy MA, Kessler RA, Rimler Z, et al. Mortality in neuromyelitis optica is strongly associated with African ancestry. Neurol Neuroinflammation. 2018;5(4):e468.
12 Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease patterns in seropositive and seronegative neuromyelitis optica: a multicentre study of 175 patients. J Neuroinflammation. 2012;9(1):14.
13 Papadopoulos MC, Bennett JL, Verkman AS. Treatment of neuromyelitis optica: state-of-the-art and emerging therapies. Nat Rev Neurol. 2014;10(9):493.
14 Mutch K, Methley A, Moore P, Jacob A. Life on hold: the experience of living with neuromyelitis optica. Disabil Rehabil. 2014;36(13):1100-1107.
15 Hinson SR, Lennon VA, Pittock SJ. Autoimmune AQP4 channelopathies and neuromyelitis optica spectrum disorders. In: Handbook of Clinical Neurology. Vol 133. Elsevier; 2016:377-403.
16 Cabrera-Gómez JA, Kurtzke JF, González-Quevedo A, Lara-Rodriguez R. An epidemiological study of neuromyelitis optica in Cuba. J Neurol. 2009;256(1):35-44.
17 Bennett JL. Finding NMO: The Evolving Diagnostic Criteria of Neuromyelitis Optica. J Neuroophthalmol. 2016;36(3):238.
18 Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Natl Acad Sci. 2012;109(4):1245-1250.
19 Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology. 2007;69(24):2221-2231.
20 Verkman AS. Aquaporins in clinical medicine. Annu Rev Med. 2012;63:303-316.
SOURCE: Alexion Pharmaceuticals
Post Views: 22
– At 48 weeks, 98% of patients treated with SOLIRIS were relapse free compared to 63% of patients receiving placebo –
– SOLIRIS is the first and only FDA approved treatment for this rare, severe, condition that attacks the central nervous system without warning –
BOSTON, MA, USA I June 27, 2019 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that 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.1 Approximately three quarters (73%) of all patients with NMOSD test positive for anti-AQP4 auto-antibodies.2 The FDA approved SOLIRIS following an expedited six-month priority review. NMOSD is a rare, severe autoimmune disease that attacks the central nervous system without warning. These attacks, also referred to as relapses, can cause progressive and irreversible damage to the brain, optic nerve and spinal cord, which may lead to long-term disability.3–6 Complement activation due to anti-AQP4 antibodies is one of the primary underlying causes of the destruction in these patients.3,7 In the PREVENT trial, SOLIRIS, a first-in-class complement inhibitor, demonstrated safety and efficacy and met its primary endpoint of prolonging the time to first adjudicated relapse and reducing the risk of relapse.1,8
“NMOSD is a serious disease with devastating consequences,” said Michael Levy, M.D., Ph.D., a consultant to the company and Associate Professor of Neurology at Massachusetts General Hospital in Boston. “Each attack can result in potentially irreversible consequences—causing blindness or losing the ability to walk—so preventing relapse is the primary goal of treatment. 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.”
NMOSD disproportionately strikes young women in the prime of their lives, with the average age of first onset at just 39 years.3 Race is also a significant risk factor for disability and mortality in NMOSD.9–11 In the U.S., African Americans are over-represented among patients diagnosed with NMOSD and more likely to suffer more frequent and more severe attacks.9–11 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.3,12,13
“Today’s approval represents an important milestone for the NMOSD community,” said Victoria Jackson, co-founder of the Guthy-Jackson Charitable Foundation (GJCF), a non-profit organization dedicated to funding research and raising awareness about NMOSD. “We are thrilled to have partnered with industry to catalyze research and development of targeted therapies to treat NMOSD. The FDA approval of SOLIRIS is the beginning of a new era for these NMOSD patients as we continue on our mission to cure this life-threatening disease.”
This approval is based on comprehensive results from the Phase 3 randomized, double-blind placebo controlled PREVENT trial, which were recently published in The New England Journal of Medicine. In the study, patients with NMOSD who were anti-AQP4 antibody positive were treated with SOLIRIS (n=96) or placebo (n=47). At 48 weeks, 98 percent of patients treated with SOLIRIS were relapse free compared to 63 percent of patients receiving placebo. This effect was observed through 144 weeks of treatment, with 96 percent of patients treated with SOLIRIS relapse free compared to 45 percent of patients in the placebo arm. SOLIRIS-treated patients experienced similar improvement in time to first adjudicated on-trial relapse with or without concomitant treatment. Of the patients treated solely with SOLIRIS, without receiving other immunosuppressive therapies (IST), (n=21), 100 percent were relapse free at 144 weeks compared to 20 percent in the placebo group (n=13).8
The safety profile of SOLIRIS was consistent with that seen for SOLIRIS in other clinical studies. 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), nasopharyngitis (21 vs. 19 percent), diarrhea (16 vs. 15 percent), back pain (15 vs. 13 percent) and dizziness (15 vs. 13 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.
“In NMOSD, disease progression is driven by attacks, and so preventing future relapses is the primary goal of treatment. Today, for the first time, adults living with anti-AQP4 antibody-positive NMOSD have an approved treatment to reduce the risk of an unpredictable relapse – or attack,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion.
The European Medicines Agency (EMA) and the Japanese Ministry of Health, Labour and Welfare (MHLW) are reviewing Alexion’s applications to add the treatment of NMOSD to the marketing authorizations for SOLIRIS in the European Union (EU) and Japan, respectively. SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with NMOSD in the U.S., EU and Japan.
About NMOSD
NMOSD is a rare and devastating 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.3,7 Patients with NMOSD experience unpredictable attacks, also referred to as relapses, which can cause irreversible damage to the brain and spinal column— and can lead to long-term disability.3–6 The most common symptoms of NMOSD are optic neuritis and transverse myelitis. Transverse myelitis can cause mobility problems including paralysis; optic neuritis can cause visual problems including blindness.14,15
Race and gender are risk factors for NMOSD.9–11 The prevalence of NMOSD may be more common and more severe in non-Caucasian populations worldwide.9–11 People of African heritage, including African Americans, are disproportionately affected compared to the general NMOSD population.9,16 The disease primarily affects women, often in the prime of their lives, with an average age of onset of 39 years.3
Approximately three quarters (73%) of all patients with NMOSD have AQP4 auto-antibodies.2 Anti-AQP4 auto-antibody testing is part of the diagnostic criteria for NMOSD.17 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. 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.13,18–20
About SOLIRIS® (eculizumab)
SOLIRIS 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. Activation of the complement cascade, plays a role in severe rare and ultra-rare disorders like paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG), and anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD). SOLIRIS is approved in the U.S., EU, Japan and other countries as a treatment for adult patients with PNH and for adults and children with aHUS. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). In the U.S., SOLIRIS is also approved for the treatment of adult patients with generalized MG (gMG) who are anti-AchR antibody-positive, in the EU as the first and only approved treatment of refractory gMG in adults who are anti-AchR antibody-positive and in Japan for the treatment of patients with gMG who are AchR antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin (IVIG) therapy or plasmapheresis (PLEX).
SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S., EU, Japan and many other countries, for the treatment of patients with aHUS in the U.S., EU and many other countries, for the treatment of patients with gMG in the U.S. for the treatment of patients with refractory gMG in EU and Japan, and for the treatment of NMOSD in the U.S., EU and Japan. Alexion and SOLIRIS have received some of the pharmaceutical industry’s highest honors for the medical innovation in complement inhibition: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
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). 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.
About Alexion
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), as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS), anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and anti-AQP4 antibody-positive 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 and metabolic disorders. Alexion has been named to the Forbes’ list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.
References
1 SOLIRIS® Product Information.
2 Hamid SHM, Whittam D, Mutch K, et al. What proportion of AQP4-IgG-negative NMO spectrum disorder patients are MOG-IgG positive? A cross sectional study of 132 patients. J Neurol. 2017;264(10):2088-2094.
3 Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG. The spectrum of neuromyelitis optica. Lancet Neurol. 2007;6(9):805-815.
4 Wingerchuk DM. Diagnosis and Treatment of Neuromyelitis Optica. Neurologist. 2007;13(1):2-11.
5 Wingerchuk DM, Weinshenker BG. Neuromyelitis optica. Curr Treat Options Neurol. 2008;10(1):55-66.
6 Kitley J, Leite MI, Nakashima I, et al. Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan. Brain. 2012;135(6):1834-1849.
7 Papadopoulos MC, Verkman AS. Aquaporin water channels in the nervous system. Nat Rev Neurosci. 2013;14(4):265.
8 Pittock SJ, Berthele A, Fujihara K, et al. Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. N Engl J Med. 2019.
9 Flanagan EP, Cabre P, Weinshenker BG, et al. Epidemiology of aquaporin‐4 autoimmunity and neuromyelitis optica spectrum. Ann Neurol. 2016;79(5):775-783.
10 Kim S-H, Mealy MA, Levy M, et al. Racial differences in neuromyelitis optica spectrum disorder. Neurology. 2018;91(22):e2089-e2099.
11 Mealy MA, Kessler RA, Rimler Z, et al. Mortality in neuromyelitis optica is strongly associated with African ancestry. Neurol Neuroinflammation. 2018;5(4):e468.
12 Jarius S, Ruprecht K, Wildemann B, et al. Contrasting disease patterns in seropositive and seronegative neuromyelitis optica: a multicentre study of 175 patients. J Neuroinflammation. 2012;9(1):14.
13 Papadopoulos MC, Bennett JL, Verkman AS. Treatment of neuromyelitis optica: state-of-the-art and emerging therapies. Nat Rev Neurol. 2014;10(9):493.
14 Mutch K, Methley A, Moore P, Jacob A. Life on hold: the experience of living with neuromyelitis optica. Disabil Rehabil. 2014;36(13):1100-1107.
15 Hinson SR, Lennon VA, Pittock SJ. Autoimmune AQP4 channelopathies and neuromyelitis optica spectrum disorders. In: Handbook of Clinical Neurology. Vol 133. Elsevier; 2016:377-403.
16 Cabrera-Gómez JA, Kurtzke JF, González-Quevedo A, Lara-Rodriguez R. An epidemiological study of neuromyelitis optica in Cuba. J Neurol. 2009;256(1):35-44.
17 Bennett JL. Finding NMO: The Evolving Diagnostic Criteria of Neuromyelitis Optica. J Neuroophthalmol. 2016;36(3):238.
18 Hinson SR, Romero MF, Popescu BFG, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Natl Acad Sci. 2012;109(4):1245-1250.
19 Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology. 2007;69(24):2221-2231.
20 Verkman AS. Aquaporins in clinical medicine. Annu Rev Med. 2012;63:303-316.
SOURCE: Alexion Pharmaceuticals
Post Views: 22
