According to a news release from biotechnology company Kira Pharmaceuticals, its therapy KP104 earned Orphan Drug designation in the United States. KP104 is in development as a therapeutic option for those living with paroxysmal nocturnal hemoglobinuria (PNH).
Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare conditions. These are defined as conditions affecting fewer than 200,000 Americans. As a benefit, drug developers – in this case, Kira Pharmaceuticals – earn incentives such as fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon approval.
So what is KP104? This bifunctional biologic, which can be administered intravenously or subcutaneously, selectively blocks both the alternative pathway and terminal pathway. This allows the therapy to target the complement system, which has been implicated in numerous diseases. In this case, KP104 targets elements within the complement system that are driving PNH development and progression.
So far, researchers have evaluated KP104 in a clinical trial with healthy volunteers; data will not be available until later in 2022. However, Kira Pharmaceuticals is moving forward with a number of different studies evaluating KP104 – not just for PNH, but for other indications such as IgA nephropathy and C3 Glomerulopathy.
What is Paroxysmal Nocturnal Hemoglobinuria (PNH)?
PIGA gene mutations cause paroxysmal nocturnal hemoglobinuria (PNH), an acquired hematopoietic stem cell (HSC) disorder. These mutations cause the proliferation of rapidly multiplying PNH cells. As the immune system attacks stem cells, healthy cells die while PNH cells survive. PIGA mutations are not inherited. Rather, they occur spontaneously.
PNH affects red and white blood cells, as well as platelets. In addition to causes red blood cell destruction (hemolysis), those with PNH may have platelet abnormalities. This is a variable disease; some individuals may survive for decades after diagnosis, while others have a shortened lifespan. Approximately 30% of PNH cases result from aplastic anemia treatment. Typically, symptoms manifest between ages 35-40. Symptoms can (but do not always) include:
- Anemia (low red blood cell count)
- Fatigue
- Chest pain
- Abdominal contractions
- Headache
- Blood clots
- Extremely pale skin
- Difficulty breathing
- Dark or bloody urine (that most often occurs late at night or early in the morning)
- Heightened heart rate
- Difficulty swallowing
- Male sexual dysfunction
- Kidney disease
