Piasky: A Novel Treatment for Paroxysmal Nocturnal Hemoglobinuria Approved on Jun 24, 2024
What is it prescribed for?
Piasky is indicated for the treatment of adult and pediatric patients 13 years and older with paroxysmal nocturnal hemoglobinuria (PNH) and body weight of at least 40 kg.
What is the name of the drug and what does it do?
Piasky (pronounced PEE-ah-skee), (generic name: crovalimab-akkz), is a medication indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adult and pediatric patients 13 years and older who weigh at least 40 kg.
PNH is a rare blood disorder that leads to the destruction of red blood cells, resulting in severe anemia, fatigue, and other complications.
How does it work?
Piasky is a complement C5 inhibitor. The complement system is part of the immune system that enhances the ability of antibodies and phagocytic cells to clear microbes and damaged cells.
It promotes inflammation and attacks the pathogen's cell membrane. In PNH, this system becomes overactive, causing the destruction of red blood cells.
Piasky specifically targets and binds to the complement protein C5.
By preventing C5 from splitting into C5a and C5b, Piasky stops the formation of the membrane attack complex (MAC). The MAC is a structure that forms on the surface of red blood cells, leading to their destruction.
By blocking this process, Piasky helps to reduce hemolysis (the breakdown of red blood cells), thus alleviating the symptoms of PNH.
What did the research discover?
Clinical trials, specifically the COMMODORE 1 and COMMODORE 2 studies, have shown that Piasky effectively controls hemolysis in PNH patients.
In these studies, patients treated with Piasky demonstrated a significant reduction in lactate dehydrogenase (LDH) levels, which is a marker of hemolysis.
LDH is an enzyme released during tissue damage, and high levels indicate the destruction of red blood cells.
Additionally, a higher proportion of patients achieved transfusion independence compared to those treated with eculizumab, the standard therapy.
Transfusion independence means patients did not require blood transfusions to manage their anemia.
Piasky also maintained hemolysis control in patients switching from other C5 inhibitors, showing it can be an effective treatment even for those previously treated with other medications.
What are some of the side effects?
- Infusion-related reactions
- Respiratory tract infections
- Viral infections
- Type III hypersensitivity reactions
What are the dosage recommendations and how is it prescribed?
Piasky is administered initially as an intravenous (IV) infusion followed by subcutaneous (SUBQ) injections. The recommended dosage regimen is as follows:
- A loading dose administered by IV infusion on Day 1
- Four additional weekly loading doses administered by SUBQ injection on Days 2, 8, 15, and 22
- A maintenance dose administered every 4 weeks by SUBQ injection starting on Day 29
The dosage is based on the patient's body weight. It is essential for healthcare providers to follow specific preparation and administration guidelines to ensure the proper and effective use of Piasky.
Source:
- Genentech, Inc. Piasky (crovalimab-akkz) injection, for intravenous or subcutaneous use. Prescribing Information. 2024. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761388s000lbl.pdf
