Recent Study Results Reveal New Possibilities in Treating Paroxysmal Nocturnal Hemoglobinuria (PNH)

On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers of paroxysmal nocturnal hemoglobinuria (PNH). Read more about this on Business Wire.

This is a disease of the blood, in which the red blood cells of the body are attacked and destroyed by the body’s own immune system. This occurs because the surface proteins on the cells are defective. These proteins would normally protect the cells from destruction. There is also an increased likelihood of blood clots, however the reason why this symptom occurs is still unknown. This dangerous symptom can lead those with the illness to an early death if left unaddressed. PNH is often discovered due to red discoloration of the urine because of cellular breakdown. To read more about this rare disease, click here.

Treatment with ALXN1210 for a period of eight months resulted in a 73 to 88 percent reduction in plasma lactate dehydrogenase, which is a direct indicator of blood cell destruction. The treatment was administered intravenously one time every eight weeks.

While ALXN1210 appears to successfully reduce the destruction of blood cells and should reduce the need for blood transfusions for those PNH, is unclear if the new product will prove itself more useful than Soliris, a treatment that acts in a similar way that was also developed by Alexion. Soliris treatments are exorbitantly costly, with a yearly treatment costing hundreds of thousands of dollars.

ALXN1210 is not expected to be a complete cure for the disease, and it does not reduce the risk of life threatening blood clots, so other treatments will be necessary to counteract the likelihood of clots. However, the testing results indicate that quality of life should nevertheless improve substantially for those with PNH. In the past, it has been estimated that one in three of those diagnosed with PNH did not survive more than five years from the time they were aware of the illness.

Additionally, the only complete cure for PNH is allogeneic bone marrow transplantation. However, this procedure often results in other significant medical problems that can be fatal on occasion. As a result, alternative treatments such as Soliris (and possibly ALXN1210 in the future) are often the only option for many people. Due to the illnesses great rarity, affecting only one or two in every million people, the development for treatment has been limited.


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