Healthcare Business

Rocket Pharma Grounded Despite Positive FDA Update

Rocket Pharmaceuticals Inc. (NASDAQ: RCKT) shares dipped on Tuesday after the company shared an update from the U.S. Food and Drug Administration (FDA). While the update wasn’t negative, this seems to be a case of sell the news, as the stock is up 65% in the past year.

The FDA granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations to RP-L102, Rocket Pharma’s lentiviral-vector-based gene therapy for the treatment of Fanconi anemia (FA).

RMAT designation was granted based on the positive efficacy and safety results from the ongoing Phase 1/2 clinical trial of RP-L102 being conducted in Europe.

Rocket plans to initiate a clinical trial of RP-L102 for FA in early 2019 utilizing no conditioning and “Process B,” which incorporates higher cell doses, transduction enhancers and commercial-grade vector manufacturing and cell processing.

Gaurav Shah, M.D., CEO and president, commented:

The RMAT designation, which is the CBER equivalent of Breakthrough Designation with the same benefits, and Fast Track designations represent important milestones for the FA program and recognition of RP-L102 as a potential treatment for this devastating disease. Achieving these milestones further underscores the urgent need to develop new treatments for young children affected by FA that would otherwise lead to bone marrow failure and death in the absence of a hematopoietic stem cell transplantation. We look forward to working closely with the FDA on our development plan to rapidly advance RP-L102 towards potential approval.

Shares of Rocket were last seen down over 6% at $15.11, in a 52-week range of $7.84 to $25.96. The consensus analyst price target is $28.00.