Alexion Receives Early FDA Approval for ULTOMIRIS™ (Ravulizumab-cwvz) in Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

- ULTOMIRIS has the potential to become the new standard of care for both complement inhibitor-naïve patients and patients receiving SOLIRIS® (eculizumab) -

BOSTON, MA, USA I December 21, 2018 I Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the U.S. Food and Drug Administration (FDA) 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 (PNH), a debilitating ultra-rare blood disorder characterized by complement-mediated destruction of the red blood cells (hemolysis). PNH can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.1,2,3,4,5,6,7,8

“We are proud to bring ULTOMIRIS to patients suffering from this devastating disease less than a year after reporting our positive Phase 3 data,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Immediate and complete C5 inhibition with ULTOMIRIS, sustained for eight weeks, can provide meaningful benefits for patients and their families. Based on the totality of our compelling data from the largest Phase 3 program ever conducted in PNH, we believe ULTOMIRIS has the potential to become the new standard of care for patients with PNH.”

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.

“We applaud the FDA’s approval of ULTOMIRIS. It is important news for patients with PNH and their families,” said Neil Horikoshi, Chief Executive Officer of the Aplastic Anemia and Myelodysplastic Syndrome (AAMDS) International Foundation. “The introduction of SOLIRIS (eculizumab) eleven years ago transformed the lives of patients with PNH. But the management of this debilitating disease still requires strength and sacrifice from patients and their families. With ULTOMIRIS, patients no longer have to plan their lives around bi-weekly infusions and can look forward to just six or seven infusions a year.”

“ULTOMIRIS is a compelling new therapy for patients with PNH. It has met the high bar for efficacy and safety established by SOLIRIS and has a four times longer dosing interval,” said Ilene Weitz, M.D., Associate Professor at the Keck School of Medicine at the University of Southern California in Los Angeles. “I am particularly pleased by the positive data in patients transitioning from SOLIRIS to ULTOMIRIS without interruption, which is critical when you treat a devastating disease like PNH. This gives me confidence in recommending that patients switch therapy.”

This approval is based on comprehensive results from two Phase 3 studies, which were recently published in Blood.9,10 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,11 and that ULTOMIRIS eliminated breakthrough hemolysis associated with incomplete C5 inhibition.12 The entire clinical development program for ULTOMIRIS to date represents more than 600 patient years of experience.

Regulatory authorities in the European Union (EU) 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.

Indication

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

Access to ULTOMIRIS

Alexion works with private healthcare organizations, policymakers and governments so that patients with rare diseases have access to the therapies they need. As part of our commitment to patients with PNH in the U.S., Alexion offers OneSource™, a personalized program that provides education, assistance with access and treatment support for patients and their caregivers. We recognize, however, that access to medicines can be challenging and are committed to supporting programs that enable eligible patients to receive our therapies.

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) 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.1,2,13 PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.1,14 PNH often goes unrecognized, 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.3,4,5,6,7,8,13 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.1,14,17 Despite historical supportive care, including transfusion and anticoagulation management, 20 to 35 percent of patients with PNH die within five to 10 years of diagnosis.18,19 Patients with certain types of hemolytic anemia, bone marrow disorders and unexplained venous or arterial thrombosis are at increased risk of PNH.13,20,21,22,23,24

About ULTOMIRIS™

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 (PNH), atypical hemolytic uremic syndrome (aHUS), and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). ULTOMIRIS is approved in the U.S. as a treatment for adults with PNH. Regulatory authorities in the European Union (EU) 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,9 and patients with PNH who had been stable on SOLIRIS (eculizumab),10 intravenous treatment with ULTOMIRIS every eight weeks demonstrated non-inferiority to intravenous treatment with SOLIRIS every two weeks on all 11 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 (gMG).

ULTOMIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and EU, and for the subcutaneous treatment of patients with aHUS in the U.S.

Please see the full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.

About Alexion

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies. As the global leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH), as well as the first and only approved complement inhibitor to treat atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG), and is also developing it for patients with neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D). In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology and metabolic disorders. Alexion has been named to the Forbes list of the World’s Most Innovative Companies seven years in a row and is headquartered in Boston, Massachusetts’ Innovation District. The company also has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

References
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SOURCE: Alexion Pharmaceuticals

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