NEW HAVEN, CT, USA I January 9, 2017 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Company has submitted marketing applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to extend the indication for Soliris® (eculizumab) as a treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. The European submission has been validated by the European Medicines Agency (EMA), marking the beginning of the review process in Europe for this potential new indication for Soliris. Both the U.S. and EU submissions are supported by the comprehensive data from the Phase 3 REGAIN study.
If approved, Soliris would address a significant unmet need for patients with refractory gMG who have largely exhausted conventional therapy. Refractory gMG patients who are anti-AChR antibody-positive are an ultra-rare segment of MG, a debilitating, complement-mediated neuromuscular disease in which patients suffer profound muscle weakness throughout the body, resulting in slurred speech, impaired swallowing and choking, double vision, disabling fatigue, shortness of breath due to respiratory muscle weakness, frequent hospital and ICU visits with prolonged stays, and episodes of respiratory failure.1-6
“Today there is an urgent need among patients suffering with refractory gMG, as there are no effective therapies for this ultra-rare and devastating disease population, causing patients to face disabling limitations in their daily lives, including difficulty walking, talking, swallowing, and breathing normally,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “The U.S. and EU regulatory submissions put us one step closer to accomplishing our goal of transforming the lives of patients suffering with refractory gMG with anti-AChR antibodies. We look forward to working with regulatory authorities as they review our applications.”
Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU. Soliris is not approved in any country for the treatment of patients with refractory gMG.
About Refractory Generalized Myasthenia Gravis
Refractory generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody-positive represent an ultra-rare segment of patients with MG—a debilitating, complement-mediated neuromuscular disease—who experience severe morbidities despite currently available MG therapies.1,2,3
MG typically begins with weakness in the ocular muscles and often progresses to the more severe and generalized form, known as gMG, to include weakness of the head, neck, trunk, limb and respiratory muscles.7 While most symptoms in gMG patients 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, resulting in slurred speech, impaired swallowing and choking, double vision, disabling fatigue, shortness of breath due to respiratory muscle weakness, frequent hospital and ICU visits with prolonged stays, and episodes of respiratory failure.4,5,6,8
Today, there are no therapies that are effective in this ultra-rare population of patients suffering from refractory gMG.
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).
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 the most robust rare disease pipeline in the biotech industry with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com.
SOURCE: Alexion Pharmaceuticals
Post Views: 63
NEW HAVEN, CT, USA I January 9, 2017 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Company has submitted marketing applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to extend the indication for Soliris® (eculizumab) as a treatment for patients with refractory generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. The European submission has been validated by the European Medicines Agency (EMA), marking the beginning of the review process in Europe for this potential new indication for Soliris. Both the U.S. and EU submissions are supported by the comprehensive data from the Phase 3 REGAIN study.
If approved, Soliris would address a significant unmet need for patients with refractory gMG who have largely exhausted conventional therapy. Refractory gMG patients who are anti-AChR antibody-positive are an ultra-rare segment of MG, a debilitating, complement-mediated neuromuscular disease in which patients suffer profound muscle weakness throughout the body, resulting in slurred speech, impaired swallowing and choking, double vision, disabling fatigue, shortness of breath due to respiratory muscle weakness, frequent hospital and ICU visits with prolonged stays, and episodes of respiratory failure.1-6
“Today there is an urgent need among patients suffering with refractory gMG, as there are no effective therapies for this ultra-rare and devastating disease population, causing patients to face disabling limitations in their daily lives, including difficulty walking, talking, swallowing, and breathing normally,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “The U.S. and EU regulatory submissions put us one step closer to accomplishing our goal of transforming the lives of patients suffering with refractory gMG with anti-AChR antibodies. We look forward to working with regulatory authorities as they review our applications.”
Soliris has received Orphan Drug Designation (ODD) for the treatment of patients with MG in the U.S. and EU. Soliris is not approved in any country for the treatment of patients with refractory gMG.
About Refractory Generalized Myasthenia Gravis
Refractory generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody-positive represent an ultra-rare segment of patients with MG—a debilitating, complement-mediated neuromuscular disease—who experience severe morbidities despite currently available MG therapies.1,2,3
MG typically begins with weakness in the ocular muscles and often progresses to the more severe and generalized form, known as gMG, to include weakness of the head, neck, trunk, limb and respiratory muscles.7 While most symptoms in gMG patients 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, resulting in slurred speech, impaired swallowing and choking, double vision, disabling fatigue, shortness of breath due to respiratory muscle weakness, frequent hospital and ICU visits with prolonged stays, and episodes of respiratory failure.4,5,6,8
Today, there are no therapies that are effective in this ultra-rare population of patients suffering from refractory gMG.
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).
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 the most robust rare disease pipeline in the biotech industry with highly innovative product candidates in multiple therapeutic areas. This press release and further information about Alexion can be found at: www.alexion.com.
SOURCE: Alexion Pharmaceuticals
Post Views: 63
