FDA Accepts Rocket Pharmaceuticals' BLA Resubmission for Rare Genetic Disorder Treatment
By Kelly Cloonan
Rocket Pharmaceuticals said the Food and Drug Administration has accepted its resubmitted Biologics License Application for Kresladi, a treatment for a rare genetic immune disorder.
The biotechnology company said Tuesday the FDA set a deadline of March 28, 2026, to decide whether to approve Kresladi.
If approved, Rocket is eligible for a Rare Pediatric Disease Priority Review Voucher, which would allow it to receive priority review from the FDA for a different product.
Kresladi aims to treat severe Leukocyte Adhesion Deficiency-I, a disorder that predisposes patients to recurrent and fatal infections. The disorder is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant, the company said.
The application is supported by positive clinical efficacy and safety data from a Phase 1/2 study of Kresladi, including fewer significant infections compared with pre-treatment levels. The data also showed evidence of improvement in skin lesions and restoration of wound-healing capabilities, the company said.
Rocket said in July it plans to cut its workforce by 30% as part of a reorganization and pipeline prioritization aimed at extending its operational runway into the second quarter of 2027. The company said at the time that it plans to focus on its AAV cardiovascular platform and on regulatory activities for Kresladi.
Write to Kelly Cloonan at kelly.cloonan@wsj.com
(END) Dow Jones Newswires
October 14, 2025 07:57 ET (11:57 GMT)
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