On March 16, 2007 the United States Food and Drug Administration (FDA) approved the only medication for PNH (paroxysmal nocturnal hemoglobinuria). PNH is a rare blood disease that affects between 8,000-10,000 individuals across Western Europe and North America.
PNH is a disease in which abnormal red blood cells are produced. When this happens a protein in the body, which is designed to kill bacteria that cause illness and infections attack these abnormal blood cells, which results in debilitating symptoms for affected individuals.
Symptoms of the condition include severe fatigue, difficulty swallowing and/or breathing, dark urine, erectile dysfunction, and severe anemia.
In the past, individuals with PNH have had to manage their symptoms with blood transfusions, supplemental therapy with iron and folic acid, anticoagulants, or steroid hormones. The only known cures for the condition were stem cell transfusions or bone marrow transfusions.
Soliris, the new medication approved by the FDA is not a medication that will cure PNH, but rather it treats the condition. The medication works by blocking the compliment system from breaking down the abnormal red blood cells caused by PNH.
The FDA conducted a double-blind clinical trial of the medication’s effectiveness on patients with PNH. The study consisted of 87 participants with PNH; some of these participants received a placebo pill while others received Soliris. The FDA discovered that half of the participants taking Soliris obtained stabilization in blood hemoglobin. On the contrary, participants who received the placebo did not achieve stabilization of blood hemoglobin.
The FDA admits that Soliris carries significant risks because it blocks the body’s immune system. Serious infections that affect the spinal cord or brain may occur while taking this medication. Meningococcal infection is among the most serious infections that can result from taking Soliris.
The FDA issued a box warning on Soliris and created a risk management plan for the medicine. They also require that all patients who take Soliris to get a meningococcal vaccine prior to taking it.
If you are affected with PNH, talk to your doctor about the risks and benefits of Soliris. He or she will be able to discuss the medication with you in detail so that you can decide together whether Soliris is an appropriate treatment option for you.
PNH is a rare blood disease that affects very few individuals in the United States. Soliris may provide new hope for the individuals that experience severe symptoms caused by the condition.