According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH).
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder that affects all three types of blood cells: red, white, and platelets. The disease can be fatal, with those surviving approximately 10 years after diagnosis. For more mild cases, individuals can survive decades after being diagnosed.
PNH destroys red blood cells and causes abnormalities in platelets. It can occur at any time, to males and females equally, and up to 30% of cases are a result of aplastic anemia treatment. The approximate median age of diagnosis is 35-40, and it affects one to two people per million.
PNH symptoms are:
- Red blood cell destruction
- Dark or bloody urine
- Increased heart rate
- Chest pain
- Difficulty breathing
- Abdomen contractions
- Difficulty swallowing
- Sexual dysfunction (men)
- Kidney disease
Empaveli, Soliris, Ultomiris
Empaveli (pegcetacoplan) is the first and only targeted C3 therapy used for treating adults living with PNH. Its approval was determined based on the results from the Phase III PEGASUS trial. The trial was 16 weeks long, and over this time Empaveli was compared to Soliris. Soliris was initially approved by the FDA as a treatment for PNH in 2007, making it the first therapy created for the condition. People within the Empaveli group had an average increase, while those a part of the Soliris group experienced a decrease.
Empaveli is to be used twice a week by a subcutaneous infusion using an infusion pump. Initially, patients will need the guidance of a health care professional, but patients and their caregivers can be trained in administering the treatment.
Soliris is given through intravenous infusion, with its doses increasing gradually over a few weeks.
Ultomiris, created by Alexion Pharmaceuticals Inc., was previously approved by the FDA. Recently, it has been approved for the treatment of children who are at least one month old or adolescents who have been diagnosed with PNH. It is the only therapy that has been approved for this age group. Ultomiris is given via an intravenous infusion similar to Soliris. However, this treatment depends on a patient’s weight, meaning it will require loading and maintenance doses.
More Options for Patients
Considering the potential for life-threatening meningococcal infections, all three drugs are available through a Risk Evaluation and Mitigation Strategy (REMS) program. Many healthcare professionals believe that having more options available for patients will aid in the management and treatment of PNH.
President and CEO of AscellaHealth, Dea Belazi, says,
“The newest therapeutic option, pegcetacoplan, provides a different mechanism of action compared to the prior therapies. Each payer will carefully review the different therapeutic options through their respective review process. In this case, pegcetacoplan is the first PNH therapy to be approved by the FDA that targets the C3 rather than C5 cell surface complement regulatory proteins.”