NEW HAVEN, CT, USA I June 23, 2017 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion to extend the current therapeutic indication for Soliris® (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. The final decision from the European Commission (EC) is anticipated in the third quarter of 2017. If approved, Soliris will be the first treatment available in the European Union (EU) for patients with refractory gMG who are anti-AChR antibody-positive, and the first and only complement inhibitor approved for this disease.
“Despite existing treatment options for gMG, patients with refractory gMG continue to suffer from severe symptoms and disease complications that significantly impact their daily lives,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “The positive CHMP opinion is a critical milestone in bringing Soliris to patients with refractory gMG who are anti-AChR antibody-positive and for whom physicians currently have no approved therapy.”
Patients with refractory gMG have difficulties walking, talking, swallowing and breathing normally. Exacerbations and crises of their disease may require hospitalization and intensive care and may be life-threatening. Patients with refractory gMG who are anti-AChR antibody-positive represent an ultra-rare segment of patients with myasthenia gravis (MG).1-10
The CHMP based its opinion on comprehensive clinical data from the Phase 3 REGAIN study (MG-301) and its long-term extension study (MG-302). A summary of the CHMP opinion can be accessed here.
Soliris is approved in the United States (U.S.), EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two ultra-rare, complement-mediated disorders. Alexion’s supplemental Biologics License Application (sBLA) in the U.S. and a supplemental new drug application in Japan for Soliris as a treatment for patients with anti-AChR antibody-positive refractory gMG have been accepted for review by the U.S. Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labour and Welfare (MHLW), respectively. Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU, and for the treatment of patients with refractory gMG in Japan.
About Refractory Generalized Myasthenia Gravis
Patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive represent an ultra-rare segment of patients with myasthenia gravis (MG) who continue to suffer from severe disease symptoms and complications despite available treatment options for gMG. There are no approved therapies for patients with anti-AChR antibody-positive refractory gMG.1-3
MG is a chronic, debilitating and progressive autoimmune neuromuscular disease that typically begins with weakness in the ocular muscles and often progresses to the more severe and generalized form, known as gMG, which includes weakness of the head, neck, trunk, limb and respiratory muscles.4-6 While most symptoms in patients with gMG are managed with conventional therapies, 10% to 15% of patients are considered refractory—meaning they do not respond to multiple conventional therapies and continue to suffer profound muscle weakness throughout the body that can result in slurred speech, impaired swallowing, double or blurred vision, disabling fatigue, shortness of breath, immobility requiring assistance, frequent hospital and intensive care unit admissions with prolonged stays and periods of respiratory failure. Complications, exacerbations and crises of refractory gMG can be life-threatening.5,7-10
In patients with anti-AChR antibody-positive MG, the body’s own immune system turns on itself to produce antibodies against AChR, a receptor located on muscle cells in the neuromuscular junction (NMJ) and used by nerve cells to communicate with the muscles these nerves control. The binding of these antibodies to AChR activates the complement cascade, another part of the immune system, which leads to progressive inflammatory damage at the NMJ. As a result, the communication between nerve and muscle is impaired, which in turn leads to a loss of normal muscle function.4,5,8,11-13
About Soliris® (eculizumab)
Soliris® is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007), European Union (2007), Japan (2010) and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. PNH is a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is also approved in the U.S. (2011), European Union (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). aHUS is a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). For the breakthrough medical innovation in complement inhibition, Alexion and Soliris have received some of the pharmaceutical industry’s highest honors: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
For more information on Soliris, please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection, available at www.soliris.net.
About Alexion
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and commercializes the first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing its rare disease pipeline with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com.
References
1. Carr A, Cardwell C, McCarron P, et al. A systemic review of population based epidemiological studies in Myasthenia Gravis. BMC Neurology. 2010, 10:46.
2. Silvestri N, Wolfe G. Treatment-refractory myathenia gravis. J. Clin Neuromuscul Dis. 2014;15(4):167-178.
3. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN.
4. Huda R, Tüzün E, Christadoss P. Targeting complement system to treat myasthenia gravis Rev. Neurosci. 2014; 25(4): 575-583
5. Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76-84.
6. Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009-8(5): 475-490.
7. Howard J. Targeting the Complement System in Refractory Myasthenia Gravis. Supplement to Neurology Reviews. February 2016.
8. National Institute of Neurological Disorders and Stroke. Myasthenia Gravis Fact Sheet. Last modified May 10, 2016. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm. Accessed May 31, 2016.
9. Sathasivam S. Diagnosis and management of myasthenia gravis. Progress in Neurology and Psychiatry. January/February 2014.
10. Howard JF. Myasthenia gravis: A manual for the healthcare provider. Myasthenia Gravis Foundation of America, Inc. 2008.
11. Conti-Fine BM, Milani M, Kaminski HJ. Myasthenia gravis: past, present, and future. J Clin Invest. 2006;116(11):2843-2854.
12. Tüzün E, Huda R, Christadoss P. Complement and cytokine based therapeutic strategies in myasthenia gravis. J Autoimmun. 2011;37(2):136-143.
13. Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev. Clin. Immunol. 2012-8(5), 427-428
SOURCE: Alexion Pharmaceuticals
Post Views: 67
NEW HAVEN, CT, USA I June 23, 2017 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion to extend the current therapeutic indication for Soliris® (eculizumab) to include the treatment of refractory generalized myasthenia gravis (gMG) in patients who are anti-acetylcholine receptor (AChR) antibody-positive. The final decision from the European Commission (EC) is anticipated in the third quarter of 2017. If approved, Soliris will be the first treatment available in the European Union (EU) for patients with refractory gMG who are anti-AChR antibody-positive, and the first and only complement inhibitor approved for this disease.
“Despite existing treatment options for gMG, patients with refractory gMG continue to suffer from severe symptoms and disease complications that significantly impact their daily lives,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “The positive CHMP opinion is a critical milestone in bringing Soliris to patients with refractory gMG who are anti-AChR antibody-positive and for whom physicians currently have no approved therapy.”
Patients with refractory gMG have difficulties walking, talking, swallowing and breathing normally. Exacerbations and crises of their disease may require hospitalization and intensive care and may be life-threatening. Patients with refractory gMG who are anti-AChR antibody-positive represent an ultra-rare segment of patients with myasthenia gravis (MG).1-10
The CHMP based its opinion on comprehensive clinical data from the Phase 3 REGAIN study (MG-301) and its long-term extension study (MG-302). A summary of the CHMP opinion can be accessed here.
Soliris is approved in the United States (U.S.), EU, Japan and other countries for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two ultra-rare, complement-mediated disorders. Alexion’s supplemental Biologics License Application (sBLA) in the U.S. and a supplemental new drug application in Japan for Soliris as a treatment for patients with anti-AChR antibody-positive refractory gMG have been accepted for review by the U.S. Food and Drug Administration (FDA) and the Japanese Ministry of Health, Labour and Welfare (MHLW), respectively. Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU, and for the treatment of patients with refractory gMG in Japan.
About Refractory Generalized Myasthenia Gravis
Patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive represent an ultra-rare segment of patients with myasthenia gravis (MG) who continue to suffer from severe disease symptoms and complications despite available treatment options for gMG. There are no approved therapies for patients with anti-AChR antibody-positive refractory gMG.1-3
MG is a chronic, debilitating and progressive autoimmune neuromuscular disease that typically begins with weakness in the ocular muscles and often progresses to the more severe and generalized form, known as gMG, which includes weakness of the head, neck, trunk, limb and respiratory muscles.4-6 While most symptoms in patients with gMG are managed with conventional therapies, 10% to 15% of patients are considered refractory—meaning they do not respond to multiple conventional therapies and continue to suffer profound muscle weakness throughout the body that can result in slurred speech, impaired swallowing, double or blurred vision, disabling fatigue, shortness of breath, immobility requiring assistance, frequent hospital and intensive care unit admissions with prolonged stays and periods of respiratory failure. Complications, exacerbations and crises of refractory gMG can be life-threatening.5,7-10
In patients with anti-AChR antibody-positive MG, the body’s own immune system turns on itself to produce antibodies against AChR, a receptor located on muscle cells in the neuromuscular junction (NMJ) and used by nerve cells to communicate with the muscles these nerves control. The binding of these antibodies to AChR activates the complement cascade, another part of the immune system, which leads to progressive inflammatory damage at the NMJ. As a result, the communication between nerve and muscle is impaired, which in turn leads to a loss of normal muscle function.4,5,8,11-13
About Soliris® (eculizumab)
Soliris® is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion. Soliris is approved in the U.S. (2007), European Union (2007), Japan (2010) and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. PNH is a debilitating, ultra-rare and life-threatening blood disorder, characterized by complement-mediated hemolysis (destruction of red blood cells). Soliris is also approved in the U.S. (2011), European Union (2011), Japan (2013) and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, or TMA (blood clots in small vessels). aHUS is a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated TMA. Soliris is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). For the breakthrough medical innovation in complement inhibition, Alexion and Soliris have received some of the pharmaceutical industry’s highest honors: the Prix Galien USA (2008, Best Biotechnology Product) and France (2009, Rare Disease Treatment).
For more information on Soliris, please see full prescribing information for Soliris, including BOXED WARNING regarding risk of serious meningococcal infection, available at www.soliris.net.
About Alexion
Alexion is a global biopharmaceutical company focused on developing and delivering life-transforming therapies for patients with devastating and rare disorders. Alexion is the global leader in complement inhibition and has developed and commercializes the first and only approved complement inhibitor to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare disorders. In addition, Alexion’s metabolic franchise includes two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing its rare disease pipeline with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com.
References
1. Carr A, Cardwell C, McCarron P, et al. A systemic review of population based epidemiological studies in Myasthenia Gravis. BMC Neurology. 2010, 10:46.
2. Silvestri N, Wolfe G. Treatment-refractory myathenia gravis. J. Clin Neuromuscul Dis. 2014;15(4):167-178.
3. Regulation (EU) No 536/2014 of the European Parliament and of the Council of 16 April 2014 on clinical trials on medicinal products for human use, and repealing Directive 2001/20/EC. http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32000R0141&qid=1421232987002&from=EN.
4. Huda R, Tüzün E, Christadoss P. Targeting complement system to treat myasthenia gravis Rev. Neurosci. 2014; 25(4): 575-583
5. Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76-84.
6. Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009-8(5): 475-490.
7. Howard J. Targeting the Complement System in Refractory Myasthenia Gravis. Supplement to Neurology Reviews. February 2016.
8. National Institute of Neurological Disorders and Stroke. Myasthenia Gravis Fact Sheet. Last modified May 10, 2016. http://www.ninds.nih.gov/disorders/myasthenia_gravis/detail_myasthenia_gravis.htm. Accessed May 31, 2016.
9. Sathasivam S. Diagnosis and management of myasthenia gravis. Progress in Neurology and Psychiatry. January/February 2014.
10. Howard JF. Myasthenia gravis: A manual for the healthcare provider. Myasthenia Gravis Foundation of America, Inc. 2008.
11. Conti-Fine BM, Milani M, Kaminski HJ. Myasthenia gravis: past, present, and future. J Clin Invest. 2006;116(11):2843-2854.
12. Tüzün E, Huda R, Christadoss P. Complement and cytokine based therapeutic strategies in myasthenia gravis. J Autoimmun. 2011;37(2):136-143.
13. Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: beyond diagnosis? Expert Rev. Clin. Immunol. 2012-8(5), 427-428
SOURCE: Alexion Pharmaceuticals
Post Views: 67
