FDA Approves Soliris for PNH Treatment

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The U.S. Food and Drug Administration (FDA) has approved Soliris (eculizumab) as the first drug for treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder defined by chronic red blood cell destruction, or hemolysis.

Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement. Soliris, the first complement inhibitor approved in the U.S. for the treatment of any disease, prevents hemolysis by selectively blocking terminal complement.

Alexion Pharmaceuticals, Inc., makers of Soliris, have also introduced Soliris OneSource, a treatment support service for all PNH patients and their healthcare providers. Enrolled patients receive support from Alexion case managers, who will provide education about PNH and Soliris and help patients obtain Soliris.

Alexion's goal is that all PNH patients who can benefit from Soliris will have access to it. Patients and their health care providers can learn more about OneSource by vistiting the website or calling 1-888-SOLIRIS (1-888-765-4747).

[Neil Cuadra]

Originally Posted by Gordman

Has it been tested so far?

Yes, it has been tested. That's necessary for FDA approval. According to the FDA announcement:

FDA based its approval on the company's randomized, double-blind, placebo-controlled clinical study of 87 patients with PNH and a series of other clinical studies. The controlled study showed that over a 26-week period half of the participants receiving Soliris had stabilization of blood hemoglobin concentrations compared with no stabilization among placebo-treated patients. Soliris-treated patients also required significantly fewer blood transfusions.

Drugs have risks as well as potential benefits, and physicians evaluate the tradeoffs for each patient. In the case of Soliris, for example, there is an increased risk of meningococcal infection, and it is recommended that patients receive meningococcal vaccinations. Minor side effects have also been reported in clinical studies, including headache, runny nose, back pain, and nausea.

PNH can be life threatening, and the availability of Soliris gives patients one more treatment option.

More information about the benefits and risks of Soliris is available from the manufacturer.

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More recently, Soliris has also been approved by the FDA to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.

Soliris’ safety and effectiveness were established in two single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 pediatric patients and 11 adult patients with aHUS. Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients with aHUS that did not respond to plasma therapy. Patients treated with Soliris also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

Soliris is manufactured by Alexion Pharmaceuticals and has been approved by the FDA to treat aHUS since 2011.