FDA Turns Away Rocket Pharma's Gene Therapy, Seeks More Data
By Colin Kellaher
Rocket Pharmaceuticals said the Food and Drug Administration has turned away the biotechnology company's application seeking approval of its proposed treatment of a rare pediatric immunodeficiency disorder due to questions related to the drug development process.
Rocket on Friday said the FDA needs limited additional information related to chemistry, manufacturing and controls to complete its review of marnetegragene autotemcel in severe leukocyte adhesion deficiency-I.
The Cranbury, N.J., company said the FDA has issued a so-called complete response letter for the gene therapy, indicating the agency won't approve the application in its current form.
The FDA earlier this year extended its target action date for a decision on marnetegragene autotemcel to June 30 after Rocket submitted clarifying chemistry, manufacturing and controls information in response to requests from the agency.
Rocket on Friday said it has met with the FDA to align on the limited scope of the additional information needed to support approval of the gene therapy, which the company plans to market as Kresladi, as quickly as possible.
Trading in shares of Rocket, which closed Thursday at $21.43, was halted premarket Friday.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
June 28, 2024 08:04 ET (12:04 GMT)
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