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EMA Reviewing Rocket Pharmaceuticals' Fanconi Anemia Gene Therapy Application

NEW YORK – The European Medicines Agency has accepted Rocket Pharmaceuticals' marketing authorization application (MAA), seeking approval for the investigational gene therapy RP-L102 as a treatment for a form of Fanconi anemia, the firm said Tuesday.

RP-L102 is a lentiviral vector-based gene therapy designed to treat a form of the rare and inherited disorder caused by mutations in the FANCA gene. The gene therapy contains patient-derived hematopoietic stem cells that have been genetically modified to contain a functional copy of the FANCA gene.

Mutations in FANCA hinder DNA repair and occur in 60 percent to 70 percent of Fanconi anemia cases. Patients with this disorder experience bone marrow failure, congenital malformations, and a predisposition to cancer.

"The acceptance of the MAA for RP-L102 marks an important step forward in our goal of bringing this potential gene therapy treatment to patients impacted by this devastating childhood disorder," Kinnari Patel, Rocket's president, head of R&D, and chief operating officer, said in a statement. "We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with FA who are in need of new treatment options."

In its regulatory application, Rocket submitted data from a global Phase I/II trial, in which RP-L102 sustained genetic and phenotypic correction and achieved hematologic stabilization for up to 42 months in patients. There were no significant safety signals and no signs of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to RP-L102.

The Cranbury, New Jersey-based firm plans to also submit a biologics license application for RP-L102 to the US Food and Drug Administration in the first half of this year.