June 24, 2024 -- The U.S. Food and Drug Administration (FDA) has approved PiaSky (crovalimab-akkz) 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.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and life-threatening blood condition in which red blood cells are destroyed by the body's complement system (part of the innate immune system). This causes symptoms such as anemia, fatigue and blood clots, and can lead to kidney disease.

Complement C5 inhibitors, which work by blocking part of the complement system cascade, have been shown to be effective in treating PNH. PiaSky contains crovalimab-akkz which is a C5 inhibitor that is recycled within the bloodstream, enabling sustained complement inhibition through low-dose administration every four weeks.

PiaSky is administered as a single loading dose by intravenous infusion on Day 1, followed by four additional weekly loading doses administered by subcutaneous injection on Days 2, 8, 15, and 22. Maintenance dosing starts on Day 29 and is administered every 4 weeks by subcutaneous injection.

The product label for PiaSky carries a Boxed Warning for an increases the risk of serious and life-threatening infections caused by Neisseria meningitidis.

PiaSky is only available through a restricted program called the PiaSky REMS.

Warnings and precautions associated with PiaSky include type III hypersensitivity reactions in patients switching from another C5 inhibitor, an increased susceptibility to serious infections, and infusion and injection-related reactions.

Common adverse reactions (incidence ≥10%) were infusion-related reaction, respiratory tract infection, viral infection, and Type III hypersensitivity reactions.

The FDA granted the approval of PiaSky to Genentech, Inc.

Posted: June 2024