Rocket Pharmaceuticals gets FDA fast track status for RP-L401 in infantile malignant osteopetrosis


Rocket Pharmaceuticals has secured fast track designation for RP-L401 gene therapy candidate from the US Food and Drug Administration (FDA) for the treatment of infantile malignant osteopetrosis (IMO).

RP-L401 is a lentiviral vector (LVV)-based gene therapy which is being studied in a non-randomized, open-label phase 1 clinical trial for the treatment of the rare childhood disorder in a couple of pediatric patients, aged one month or older. It was in-licensed by the US biotech company from Sweden-based Lund University.

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Infantile malignant osteopetrosis is a severe monogenic bone resorption disorder that is characterized by skeletal deformities, neurologic abnormalities, and bone marrow failure, said Rocket Pharmaceuticals.

Kinnari Patel – Chief Operating Officer and Executive Vice President, Development at Rocket Pharmaceuticals said: “We are proud to announce Rocket’s fifth Fast Track designation, an accomplishment that truly embodies our mission and vision of bringing curative gene therapies to children suffering from rare disorders.

“Advancing five programs into the clinic in five years is a testament that we are on our way towards achieving that mission. IMO is one of the most devastating pediatric disorders, and we are grateful to our team and our collaborators for all of the work that they’ve done in moving this program forward for children and their families.”

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Rocket Pharmaceuticals said that the fast track designation will give the company greater access to the FDA for the purpose of accelerating RP-L401’s development, review, and potential approval.

Apart from that, the fast track status gives the gene therapy candidate eligibility for the FDA’s accelerated approval and priority review, should it meet relevant criteria, besides rolling review, which allows Rocket Pharmaceuticals to submit completed portions of its biologic license application (BLA) for review by FDA, instead of waiting until every section is completed before the full application can be reviewed by the regulator.

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