Therapy Areas: Vaccines
Alexion Receives Early FDA Approval for Ultomiris in Adults with Paroxysmal Nocturnal Hemoglobinuria
26 December 2018 - - The US Food and Drug Administration has approved Ultomiris (ravulizumab-cwvz), the first and only long-acting C5 complement inhibitor administered every eight weeks, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria, a debilitating ultra-rare blood disorder characterized by complement-mediated destruction of the red blood cells (hemolysis), US-based Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) said.

PNH can cause a range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.

The approval of Ultomiris comes ahead of the Prescription Drug User Fee Act (PDUFA) date of February 18, 2019, set by the FDA as part of an expedited eight month review following Alexion's use of a rare disease priority review voucher.

This approval is based on comprehensive results from two Phase 3 studies, which were recently published in Blood.

In these studies, which included 441 patients who had either never been treated with a complement inhibitor before, or who had been stable on SOLIRIS, the efficacy of ULTOMIRIS administered every eight weeks was non-inferior to the efficacy of SOLIRIS administered every two weeks on all 11 endpoints.

The safety profile of ULTOMIRIS was similar to that of SOLIRIS. Recently presented additional data showed that Ultomiris provided immediate and complete C5 inhibition that was sustained for eight weeks, and that Ultomiris eliminated breakthrough hemolysis associated with incomplete C5 inhibition.

The entire clinical development program for ULTOMIRIS to date represents more than 600 patient years of experience.

Regulatory authorities in the European Union and Japan have accepted and are reviewing applications for the approval of ULTOMIRIS as a treatment for adults with PNH. Alexion continues to work with the authorities to enable timely reviews.

Ultomiris is a prescription medicine called a monoclonal antibody. ULTOMIRIS is used to treat adult patients with a disease called Paroxysmal Nocturnal Hemoglobinuria.

Paroxysmal nocturnal hemoglobinuria is a chronic, progressive, debilitating and life-threatening ultra-rare blood disorder characterized by hemolysis (destruction of red blood cells) that is mediated by an uncontrolled activation of the complement system, a component of the body's immune system.

PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.

PNH often goes unrecognised, with delays in diagnosis ranging from one to more than five years.15 Patients with PNH may experience a wide range of signs and symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia.

The most devastating consequence of chronic hemolysis is thrombosis, which can occur in blood vessels throughout the body, damage vital organs and cause premature death.16 The first thrombotic event can be fatal.

Despite historical supportive care, including transfusion and anticoagulation management, 20 to 35 % of patients with PNH die within five to 10 years of diagnosis.

Patients with certain types of hemolytic anemia, bone marrow disorders and unexplained venous or arterial thrombosis are at increased risk of PNH.

Ultomiris (ravulizumab-cwvz) is the first and only long-acting C5 inhibitor administered every eight weeks that works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, and anti-acetylcholine receptor antibody-positive myasthenia gravis.

Ultomiris is approved in the US as a treatment for adults with PNH. Regulatory authorities in the European Union and Japan have accepted and are reviewing applications for the approval of ULTOMIRIS as a treatment for adults with PNH.

In Phase 3 clinical studies in complement inhibitor-naïve patients with PNH, and patients with PNH who had been stable on Soliris (eculizumab), intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all endpoints.

Ultomiris is also currently being evaluated in a Phase 3 clinical study in complement inhibitor-naïve patients with aHUS, administered intravenously every eight weeks.

In addition, Alexion plans to initiate a Phase 3 clinical study of Ultomiris delivered subcutaneously once per week as a potential treatment for patients with PNH and aHUS.

Alexion is also planning to initiate the development of Ultomiris, intravenously administered every eight weeks, as a potential treatment for patients with generalized MG.

Ultomiris has received Orphan Drug Designation for the treatment of patients with PNH in the US and EU, and for the subcutaneous treatment of patients with aHUS in the US.

Ultomiris is a prescription medicine called a monoclonal antibody. Ultomiris is used to treat adults with a disease called Paroxysmal Nocturnal Hemoglobinuria. It is not known if Ultomiris is safe and effective in children.

Ultomiris is a medicine that affects the immune system. Ultomiris can lower the ability of the immune system to fight infections. Ultomiris increases the chance of getting serious and life-threatening meningococcal infections.

Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.

Meningococcal vaccines must be received at least 2 weeks before the first dose of ULTOMIRIS if one has not already had this vaccine.

If one's doctor decided that urgent treatment with ULTOMIRIS is needed, meningococcal vaccination should be administered as soon as possible.
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