The FDA has recently approved a treatment called Pyrukynd as a therapeutic option for hemolytic anemia disorder sparked by pyruvate kinase deficiency.
Hemolytic anemia is a condition where red blood cells are killed faster than they can be produced. This new therapeutic option works to increase the concentration of hemoglobin.
PK Deficiency
PK deficiency is a rare disease which is often misdiagnosed. It is also suspected that the condition is highly undiagnosed.
The condition destroys red blood cells prematurely and ultimately leads to ongoing anemia.
Symptoms of PK Deficiency include jaundice, fatigue, a rapid heart rate, shortness of breath, and pale skin. More severe symptoms include gallstones and an enlarged spleen. Some patients may face an overcorrection of anemia and ultimately high iron levels from repeated blood transfusions.
Effectiveness
Pyrukynd has already received Fast Track Designation, Priority Review, and Orphan Drug Designation.
Its FDA approval comes from 2 clinical studies. The first was a randomized study, and the second was a single arm study.
Randomized Study
The first study was a randomized and placebo controlled investigation including 80 adult PK deficiency patients who had not received regular blood transfusions. Patients who were in the treatment group received 50mg of the therapy orally twice each day. The treatment was administered for 24 weeks.
The primary endpoint of this trial was an increase in the concentration of hemoglobin of 1.5 g/dL or more. Researchers hoped to see this increase sustained for a minimum of 2 assessments.
This trial found that 40% of those in the treatment group experienced a hemoglobin response. No participants in the placebo group experienced a hemoglobin response.
Single Arm Study
The second study was a single arm investigation including 27 adult patients who had all been receiving regular blood transfusions. The dosage of Pyrukynd in this trial was the same as the randomized investigation, but in this study treatment was given for 40 weeks instead of 24.
In this study, the primary endpoint was a 33% reduction in transfusion burden over 24 weeks.
33% of all participants met the primary endpoint. Additionally, 22% of all participants didn’t require blood transfusions following the 24 week treatment period.
There were some side effects associated with this therapy. For males, decreases in estradiol and estrone were common. Levels of these hormones in females were more difficult to assess due to the natural fluctuations in this hormone that they experience. Additionally, both sexes commonly faced an increase in urate, joint pain, and back pain.
You can read more about these studies and this drugs approval here.
