FDA Approves Therapy for Rare Disorder Affecting Red Blood Cells

The U.S. Food and Drug Administration (FDA) has approved Fabhalta® (iptacopan), the first oral therapy for the treatment of adults with a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). It is manufactured by Novartis.

PNH is an acquired (not inherited) condition that causes the immune system to attack and prematurely breakdown red blood cells and platelets. Left untreated, the condition can become serious and even be life threatening. While it can occur at any age, it is most often diagnosed in young adults.

Symptoms of the disorder range from mild-to-severe and can include fatigue, dizziness, chronic kidney disease, abdominal pain, excessive blood clots, and hemolytic anemia (HA).

HA happens when red blood cells are destroyed faster than they can be produced. Because red blood cells carry the oxygen transport protein hemoglobin, a spike in their destruction causes high concentrations of hemoglobin. The results of HA are apparent in the dark red or brown coloring of an affected individual’s urine – this most often occurs at night or in the early morning.

Individuals with PNH often need red blood cell transfusions.

There are other PNH treatments that work differently and are administered intravenously. Fabhalta®, which is taken via an oral capsule, helps limit the destruction of red blood cells. Read the company release to learn more about the new therapy, including the clinical trials that contributed to its FDA approval.

“An efficacious oral treatment with a demonstrated safety profile could be practice-changing for physicians and help relieve burdens experienced by people with PNH,” said Vinod Pullarkat, MD, MRCP, Clinical Professor, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope.

Source: Novartis news release dated December 6, 2023