NEW YORK – Rocket Pharmaceuticals on Thursday said it has initiated a rolling biologics license application with the US Food and Drug Administration for RP-L102, its investigational gene therapy candidate for Fanconi anemia.
RP-L102 is a lentiviral vector-based gene therapy that Rocket is developing to treat a form of Fanconi anemia, a rare and inherited disorder that can be 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.
The European Medicines Agency earlier this year accepted Rocket's marketing authorization application seeking approval for RP-L102, and review of the gene therapy is underway.
The firm also said it had secured an International Classification of Diseases-10 code from the US Centers for Medicare & Medicaid Services, which will allow physicians and others to better identify patients diagnosed with Fanconi anemia and track the care they are receiving in the healthcare system.
Rocket provided the regulatory update on RP-L102 on Thursday in conjunction with reporting its third quarter 2024 financials. The firm, which doesn't have any revenue-generating products, posted a $66.7 million net loss, or $.71 per share, in Q3 2024, compared to a net loss of $61.9 million, or $.75 per share, in the year-ago quarter.
As of Sept. 30, the Cranbury, New Jersey-based firm had $235.7 million in cash, cash equivalents, and investments, which it expects will fund operations into 2026.