Rocket Pharma to light up buildings again for Rare Disease Day as it awaits first FDA decision
26 Feb 2024
Priority ReviewGene Therapy
Rocket Pharma is getting ready to light up national landmarks on Thursday as part of its annual rare disease awareness effort, but this year the event is even more meaningful as Rocket awaits an FDA decision on its first drug candidate.
Rocket’s gene therapy candidate, Kresladi, to treat severe leukocyte adhesion deficiency-I (LAD-I) is being considered under priority review with a June 30 decision date. Rocket also plans to submit another biologics license application (BLA) in the first half of the year for RP-L102 treatment for fanconi anemia.
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Source: EndPoints
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