A Treatment for Paroxysmal Nocturnal Hemoglobinuria Was Accepted for Priority Review

According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their experimental treatment ALXN1210. This drug is in development for the treatment of paroxysmal nocturnal hemoglobinuria. Alexion also opted to used a priority review voucher for the drug.

About PNH

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life threatening illness in which the body’s complement system, which is a component of the immune system, begins to destroy red blood cells. The disease is caused by an acquired defect of the red blood cell membrane, which would prevent an attack from the immune system in health people. It is often linked to bone marrow disorders. Symptoms of PNH include red urine, palpitations, fatigue, shortness of breath, painful and difficult swallowing, erectile dysfunction, blood clots, and abdominal pain. This long-term disease can appear in episodes of acute symptoms. Treatment options are limited, but there are options available to reduce blood clots and and improve overall quality of life. Patient survival after diagnosis and without disease modifying care ranges from 10-20 years. To learn more about PNH, click here.

Accelerated Approval?

With the use of priority review, the FDA will review the application over an 8 month period instead of the typical 12 month review. The decision to accept priority review and the Biologics Licensing Application are rooted in a robust trove of data collected in a large scale Phase III clinical trial. This trial involved a total of 440 patients.

A New Opportunity for PNH Patients

If ALXN1210 succeeds in gaining approval, it will become the first treatment that can offer long-term relief for patients, with dosing occurring at eight week intervals. The current standard of care, eculizumab, can only achieve the same results with two week dosing intervals. Clearly, this experimental therapy has the potential to offer improved quality of life for patients. ALXN1210 is also under review by the European Medicines Agency for treating PNH, and Alexion plans to submit an application in Japan soon as well. ALXN1210 has also received Orphan Drug designations in both the US and the EU.


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