First and Only Approved Treatment in both Europe and the United States for Patients with aHUS, an Ultra-Rare, Life-Threatening Disease
CHESHIRE, CT, USA I November 29, 2011 I Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that the European Commission (EC) has extended the therapeutic indication for Soliris® (eculizumab) to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS). Soliris is the first therapy approved in the European Union for the treatment of aHUS, an ultra-rare, life-threatening, chronic, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.1
The morbidity and premature mortality in aHUS are caused by chronic uncontrolled activation of the complement system, resulting in the formation of multiple blood clots in small blood vessels throughout the body, known as thrombotic microangiopathy or TMA.2,3 Despite historical supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4
"The approval of Soliris for aHUS in Europe is a major milestone for patients with aHUS, whose uncontrolled complement activation leads to progressive organ failure and a broad range of life-threatening outcomes," said Christophe Legendre, M.D., Professor of Nephrology at University Rene Descartes-Hôpital Necker in Paris.5 "In clinical studies, chronic treatment with Soliris resulted in a rapid and sustained reduction in complement-mediated TMA. This drug alters the course of aHUS and can make a dramatic difference in patients’ lives."
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation. The EC has granted marketing authorization for Soliris to treat pediatric and adult patients with aHUS. The Clinical Particulars section of the EU label states, "Soliris treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of Soliris is clinically indicated," as described in the Special warnings and precautions for use subsection. Alexion will begin reimbursement discussions with healthcare authorities in major European countries, and expects to start serving patients with aHUS in initial major European countries in the first half of 2012, with additional major European countries commencing through mid-2013.
"The EC approval marks another milestone for Soliris and brings life-transforming hope another step closer to families in Europe living with this severe, devastating and life-threatening disease," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We will work diligently with the healthcare authorities in individual countries to make Soliris available to children and adults with aHUS as quickly as possible."
Soliris was previously approved by the U.S. Food and Drug Administration (FDA) on September 23, 2011 for the treatment of patients with aHUS to inhibit complement-mediated TMA. Soliris is also approved in the United States, European Union, Japan and other territories for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder.
Soliris in aHUS Clinical Data
The approval of Soliris for the treatment of aHUS is based on clinical data from two prospective, controlled, open-label studies in adolescent and adult patients with aHUS, and a third retrospective study in pediatric patients with aHUS, which together included a broad range of aHUS patients. All patients treated with Soliris when administered as recommended demonstrated rapid and sustained reduction in terminal complement activity, and chronic administration of Soliris resulted in rapid and sustained reduction in complement-mediated TMA.
In study C08-003 A/B, which included 20 patients with a long duration of aHUS and prior treatment before starting on Soliris,16 out of 20 Soliris-treated patients (80%) achieved TMA event-free status, defined as at least 12 consecutive weeks of stable platelet count, no plasma exchange/plasma infusion (PE/PI), and no new dialysis. Hematologic normalization was achieved in 18 of 20 Soliris-treated patients (90%). Renal function, as measured by eGFR, increased during Soliris therapy, and no patient required new dialysis.
In study C08-002 A/B, which included 17 patients with progressive clinical TMA complications despite intensive PE/PI, Soliris inhibited complement-mediated TMA as shown by a significant improvement in platelet count from baseline through week 26 of 73×109/L (p=0.0001). Hematologic normalization was observed in 13 of 17 Soliris-treated patients (76%), and TMA event-free status (stable platelet count, no PE/PI, and no new dialysis) was achieved by 15 of 17 Soliris-treated patients (88%). Renal function, as measured by eGFR, was significantly improved during Soliris therapy, and four out of five patients who required dialysis at study entry were able to discontinue dialysis for the duration of treatment. Patients also reported improved health-related quality of life.
Study C009-001r included 15 pediatric patients (ages 2 months to <12 years) who received Soliris outside of prospective clinical trials and with or without prior PE/PI. Platelet count was normalized in 14 of 15 Soliris-treated patients (93%). All patients treated with Soliris also achieved a reduction in the TMA intervention rate. The efficacy results for these pediatric patients appeared consistent with results for patients enrolled in the prospective aHUS studies. No pediatric patient required new dialysis during treatment with Soliris.
Soliris was well tolerated in these clinical studies. The most frequently reported adverse events were hypertension, upper respiratory tract infection, and diarrhea.
About aHUS
aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes life-long uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.1,2 Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death. 2,3 More than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4 The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.6
aHUS affects both children and adults. In a large group of aHUS patients, 60% were first diagnosed at younger than 18 years of age.6 Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.7
About Soliris
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 US, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), 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 US as the first and only treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated thrombotic microangiopathy (blood clots in small vessels). The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Alexion’s breakthrough approach in complement inhibition has received the pharmaceutical industry’s highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases. More information including the full prescribing information on Soliris is available at www.soliris.net.
SOURCE: Alexion Pharmaceuticals
Post Views: 75
First and Only Approved Treatment in both Europe and the United States for Patients with aHUS, an Ultra-Rare, Life-Threatening Disease
CHESHIRE, CT, USA I November 29, 2011 I Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that the European Commission (EC) has extended the therapeutic indication for Soliris® (eculizumab) to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS). Soliris is the first therapy approved in the European Union for the treatment of aHUS, an ultra-rare, life-threatening, chronic, genetic disease that progressively damages vital organs, leading to stroke, heart attack, kidney failure and death.1
The morbidity and premature mortality in aHUS are caused by chronic uncontrolled activation of the complement system, resulting in the formation of multiple blood clots in small blood vessels throughout the body, known as thrombotic microangiopathy or TMA.2,3 Despite historical supportive care, more than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4
"The approval of Soliris for aHUS in Europe is a major milestone for patients with aHUS, whose uncontrolled complement activation leads to progressive organ failure and a broad range of life-threatening outcomes," said Christophe Legendre, M.D., Professor of Nephrology at University Rene Descartes-Hôpital Necker in Paris.5 "In clinical studies, chronic treatment with Soliris resulted in a rapid and sustained reduction in complement-mediated TMA. This drug alters the course of aHUS and can make a dramatic difference in patients’ lives."
Soliris, a first-in-class terminal complement inhibitor, specifically targets uncontrolled complement activation. The EC has granted marketing authorization for Soliris to treat pediatric and adult patients with aHUS. The Clinical Particulars section of the EU label states, "Soliris treatment is recommended to continue for the patient’s lifetime, unless the discontinuation of Soliris is clinically indicated," as described in the Special warnings and precautions for use subsection. Alexion will begin reimbursement discussions with healthcare authorities in major European countries, and expects to start serving patients with aHUS in initial major European countries in the first half of 2012, with additional major European countries commencing through mid-2013.
"The EC approval marks another milestone for Soliris and brings life-transforming hope another step closer to families in Europe living with this severe, devastating and life-threatening disease," said Leonard Bell, M.D., Chief Executive Officer of Alexion. "We will work diligently with the healthcare authorities in individual countries to make Soliris available to children and adults with aHUS as quickly as possible."
Soliris was previously approved by the U.S. Food and Drug Administration (FDA) on September 23, 2011 for the treatment of patients with aHUS to inhibit complement-mediated TMA. Soliris is also approved in the United States, European Union, Japan and other territories for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating, ultra-rare and life-threatening blood disorder.
Soliris in aHUS Clinical Data
The approval of Soliris for the treatment of aHUS is based on clinical data from two prospective, controlled, open-label studies in adolescent and adult patients with aHUS, and a third retrospective study in pediatric patients with aHUS, which together included a broad range of aHUS patients. All patients treated with Soliris when administered as recommended demonstrated rapid and sustained reduction in terminal complement activity, and chronic administration of Soliris resulted in rapid and sustained reduction in complement-mediated TMA.
In study C08-003 A/B, which included 20 patients with a long duration of aHUS and prior treatment before starting on Soliris,16 out of 20 Soliris-treated patients (80%) achieved TMA event-free status, defined as at least 12 consecutive weeks of stable platelet count, no plasma exchange/plasma infusion (PE/PI), and no new dialysis. Hematologic normalization was achieved in 18 of 20 Soliris-treated patients (90%). Renal function, as measured by eGFR, increased during Soliris therapy, and no patient required new dialysis.
In study C08-002 A/B, which included 17 patients with progressive clinical TMA complications despite intensive PE/PI, Soliris inhibited complement-mediated TMA as shown by a significant improvement in platelet count from baseline through week 26 of 73×109/L (p=0.0001). Hematologic normalization was observed in 13 of 17 Soliris-treated patients (76%), and TMA event-free status (stable platelet count, no PE/PI, and no new dialysis) was achieved by 15 of 17 Soliris-treated patients (88%). Renal function, as measured by eGFR, was significantly improved during Soliris therapy, and four out of five patients who required dialysis at study entry were able to discontinue dialysis for the duration of treatment. Patients also reported improved health-related quality of life.
Study C009-001r included 15 pediatric patients (ages 2 months to <12 years) who received Soliris outside of prospective clinical trials and with or without prior PE/PI. Platelet count was normalized in 14 of 15 Soliris-treated patients (93%). All patients treated with Soliris also achieved a reduction in the TMA intervention rate. The efficacy results for these pediatric patients appeared consistent with results for patients enrolled in the prospective aHUS studies. No pediatric patient required new dialysis during treatment with Soliris.
Soliris was well tolerated in these clinical studies. The most frequently reported adverse events were hypertension, upper respiratory tract infection, and diarrhea.
About aHUS
aHUS is a chronic, ultra-rare, and life-threatening disease in which a genetic deficiency in one or more complement regulatory genes causes life-long uncontrolled complement activation, resulting in complement-mediated thrombotic microangiopathy (TMA), the formation of blood clots in small blood vessels throughout the body.1,2 Permanent, uncontrolled complement activation in aHUS causes a life-long risk for TMA, which leads to sudden, catastrophic, and life-threatening damage to the kidney, brain, heart, and other vital organs, and premature death. 2,3 More than half of all patients with aHUS die, require kidney dialysis or have permanent kidney damage within 1 year of diagnosis.4 The majority of patients with aHUS who receive a kidney transplant commonly experience subsequent systemic TMA, resulting in a 90% transplant failure rate.6
aHUS affects both children and adults. In a large group of aHUS patients, 60% were first diagnosed at younger than 18 years of age.6 Complement-mediated TMA also causes reduction in platelet count (thrombocytopenia) and red blood cell destruction (hemolysis). While mutations have been identified in at least ten different complement regulatory genes, mutations are not identified in 30-50% of patients with a confirmed diagnosis of aHUS.7
About Soliris
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 US, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), 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 US as the first and only treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy, a debilitating, ultra-rare and life-threatening genetic disorder characterized by complement-mediated thrombotic microangiopathy (blood clots in small vessels). The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). Alexion’s breakthrough approach in complement inhibition has received the pharmaceutical industry’s highest honors: the 2008 Prix Galien USA Award for Best Biotechnology Product with broad implications for future biomedical research and the 2009 Prix Galien France Award in the category of Drugs for Rare Diseases. More information including the full prescribing information on Soliris is available at www.soliris.net.
SOURCE: Alexion Pharmaceuticals
Post Views: 75
