The U.S. Food and Drug Administration approved Soliris (eculizumab) to treat individuals with atypical hemolytic Uremic Syndrome (aHUS). aHUS is a rare and chronic blood disease that can result in kidney (renal) failure and is linked with an increased risk of death and stroke.
This disease accounts for 5 to 10% of all cases of hemolytic uremic syndrome. In the vast majority of cases atypical HUS only affects children.
Soliris is a targeted treatment that operates by restricting proteins that play a role in aHUS. In 2007 the FDA initially approved this drug to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare type of blood disorder that can result in disability and premature death. Soliris is classified as an orphan drug. Orphan drugs are medications that show potential for the diagnosis and/or treatment of rare diseases or conditions.
No other treatments for aHUS have been FDA-approved, and the safety and efficiency of current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been investigated in well managed trials.
Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, explained:
"This is the first approval of a drug for treating this life-threatening disease, and the first approval for use of Soliris in children. This approval underscores how an increased understanding of the biology of a disease and of how a drug interacts with that process can expedite drug development."
Two single-arm trials that consisted of 37 adults and adolescent individuals with aHUS and one retrospective investigation involving 19 pediatric patients and 11 adult patients with aHUS, were conduced to establish the safety and effectiveness of Soliris. The participants on Soliris experienced improvements in kidney function, including elimination of the requirement for dialysis in several individuals with aHUS for whom plasma therapy was not effective. The Soliris participants also showed improvement in platelet counts and other blood parameters that relate with aHUS disease activity.
Among those on Soliris, the most common adverse events included high blood pressure (hypertension), diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and a decrease in white blood cells (leukopenia).
This new evidence for Soliris is being approved with an extension of the existing Risk Evaluation and Mitigation Strategy (REMS), in order to let health care professionals and patients know about the known risk of life-threatening meningococcal infections.
Access to Soliris will be restricted to those whose doctors have been enrolled in a registration program. The physician will have to provide the patient with a guide.
The Soliris submission was reviewed under the FDA's priority review program. This accelerated six-month review is reserved for medications that may provide considerable advances in treatment, or in cases where new treatment can be provided when no adequate one currently exists.
In addition, the treatment is being approved under the FDA's accelerated approval program, designed to offer patients faster access to promising new drugs followed by further investigations to confirm the drug's clinical benefit.
The accelerated approval program allows the agency to approve a drug to treat a serious disease based on clinical data demonstrating that the medication has an effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, or on an effect on a clinical endpoint other than survival or irreversible morbidity.
Soliris is marketed by Alexion Pharmaceuticals in Cheshire, Conn.
Written by Grace Rattue