Alexion Submits Application in Japan for Soliris for Patients with Refractory Generalized Myasthenia Gravis.
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- US-based biopharmaceutical company Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) has submitted an application to Japan's Ministry of Labour and Welfare to extend the indication for Soliris (eculizumab) as a potential treatment for patients with refractory generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive, the company said.
The submission is supported by comprehensive data from the Phase 3 REGAIN study.
Patients with AChR-positive refractory gMG represent an ultra-rare population. Despite existing treatment options for gMG, these patients continue to face severe complications, including difficulty walking, talking, swallowing, and breathing normally.
Exacerbations of their disease may be life-threatening and require hospitalisation and intensive care.
If approved, Soliris would be the first and only complement inhibitor for patients with refractory AChR-positive gMG in Japan.
Alexion has also submitted marketing applications in the US and EU to extend the indication for Soliris as a potential treatment for patients with refractory gMG who are AChR-positive, which have been accepted by the US Food and Drug Administration and validated by the European Medicines Agency.
Soliris has received Orphan Drug Designation for the treatment of patients with MG in the US and EU, and for treatment of patients with refractory gMG in Japan.
It is not approved in any country for the treatment of patients with refractory AChR-positive gMG.
Refractory generalized myasthenia gravis patients who are anti-acetylcholine receptor antibody-positive represent an ultra-rare segment of patients with MG, a chronic, debilitating and progressive autoimmune neuromuscular disease where the complement system mediates a progressive, destructive inflammatory effect on the neuromuscular junction.
Patients with refractory AChR-positive gMG experience severe morbidities and life-threatening complications despite currently available MG therapies.
MG typically begins with weakness in the ocular muscles and often progresses to the more severe and generalised form, known as gMG, to include weakness of the head, neck, trunk, limb and respiratory muscles.
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 and choking, double vision, disabling fatigue, shortness of breath due to respiratory muscle weakness, immobility requiring assistance, frequent hospital and intensive care unit admissions with prolonged stays, and periods of respiratory failure.
In patients with AChR-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 and used by nerve cells to communicate with the muscles these nerves control.
The binding of these antibodies to AChR activates the complement cascade which leads to the destruction of the NMJ. As a result, the communication between nerve and muscle is disrupted, which leads to a loss of normal muscle function.
Currently, there are no approved therapies for the ultra-rare population of patients suffering from refractory AChR-positive gMG.
Soliris is a first-in-class terminal complement inhibitor developed from the laboratory through regulatory approval and commercialization by Alexion.
It is approved in the US, European Union, Japan and other countries as the first and only treatment for patients with paroxysmal nocturnal hemoglobinuria to reduce hemolysis.
PNH is a debilitating, ultra-rare and life-threatening blood disorder, characterised by complement-mediated hemolysis (destruction of red blood cells).
Soliris is also approved in the US, European Union, Japan and other countries as the first and only treatment for patients with atypical hemolytic uremic syndrome 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).
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|Date:||Mar 23, 2017|
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