FDA Again Delays Approval of Rocket Pharmaceuticals’ Gene Therapy Treatment

Rocket Pharmaceuticals (RCKT) shares declined in intraday trading Friday after the biopharmaceutical firm announced that the Food and Drug Administration (FDA) wants more data about its gene therapy to treat a rare immune disorder in children before granting approval.

The company reported that the FDA sent it a Complete Response Letter (CRL) asking for “limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review” of the drug, called Kresladi.

Rocket noted that it has met with senior leadership of the FDA’s Center for Biologics Evaluation and Research (CBER) to determine what is needed. Chief Executive Officer (CEO) Dr. Gaurav Shah said he was hopeful of a quick resolution because of “CBER leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients.”

FDA Initially Delayed Kresladi Decision Earlier This Year

This is the second time the FDA has delayed a decision on Kresladi, which is designed to treat severe leukocyte adhesion deficiency-I (LAD-I). Rocket had anticipated action by the end of March, but officials extended that by three months, also because of questions about CMC.

Analysts at William Blair noted that while they were disappointed at the issuing of the CRL, they were confident that the company will quickly meet the request and resubmit Kresladi “in a timely manner.” Blair has an “outperform” rating on the stock. 

Shares of Rocket Pharmaceuticals were down 1.3% to $21.15 as of 12:30 p.m. ET Friday, and have lost nearly 30% of their value year-to-date.