Rocket Pharmaceuticals received accelerated approval from the U.S. Food and Drug Administration for its gene therapy Kresladi, intended to treat leukocyte adhesion deficiency-I, according to a March 27 announcement. The decision follows a previous rejection in 2024 due to manufacturing concerns.
Leukocyte adhesion deficiency-I is a rare immune disorder that leads to frequent and severe infections, affecting about one in every million people worldwide. Analysts at Jefferies estimate that this represents a small market opportunity with projected sales below $50 million, and they expect fewer than ten patients per year will be treated due to the rarity of the disease.
Jefferies analysts said, “Kresladi’s US launch provides RCKT with experience in payor negotiations and commercial scaling, which can be leveraged for future launches.” They also noted that the approval reduces risk for Rocket’s gene therapy platform and manufacturing network, potentially making future regulatory reviews smoother.
The FDA based its decision on pivotal Phase 1/2 data from October 2023 showing a 100% overall response rate at twelve months. Common side effects included anemia, low platelet counts, upper respiratory infections, and elevated liver enzymes. The agency granted accelerated approval with full approval dependent on confirmation of clinical benefit in an additional trial.
With Kresladi now approved, attention turns to Rocket’s lead asset RP-A501 for Danon disease. Last May, the FDA placed this program under clinical hold after a patient died from complications related to capillary leak syndrome following treatment. The hold was lifted in August after adjustments were made to dosing protocols. Jefferies expects Rocket will restart its Phase 2 Danon study during the first half of 2026.
