RCKT - Rocket Pharmaceuticals: CRL For Kresladi Unwelcome But Likely A Quick Fix

2024-06-28 12:08:19 ET

Summary

• Rocket Pharmaceuticals stock dropped pre-market after management reported receiving a Complete Response Letter from the FDA in relation to Kresladi, indicated for severe leukocyte adhesion deficiency-I.
• The rejection was based on Chemistry Manufacturing and Controls issues, which can typically be addressed quickly, suggesting a good chance of approval upon resubmission.
• Rocket's delay in approval for Kresladi should not impact its overall investment opportunity, with a diverse pipeline and potentially valuable assets in its portfolio.
• The key to the company's fortunes is its in vivo therapy targeting Danon Disease. Unless the CMC issues identified by the FDA seriously affect Rocket's manufacturing plant, causing delays, today's setback should not be viewed as too problematic.

Investment Overview

Rocket Pharmaceuticals ( RCKT ) stock was tanking in pre-market trading today, after the Cranbury, N.J.,-based biotech reported it had received a Complete Response Letter ("CRL") from the Food and Drug Administration, rejecting the company's Biologics License Application ("BLA") for its lentiviral ("LV") vector-based gene therapy Kresladi to treat severe leukocyte adhesion deficiency-I (LAD-I)....

For further details see: