Guess the Therapy Answer January 21, 2026

Which investigational genetically modified autologous hematopoietic stem cell therapy candidate for leukocyte adhesion deficiency type 1 (LAD-I) has an upcoming PDUFA date for March 28, 2026?

Answer: marnetegragene autotemcel (RP-L201, to be marketed as Kresladi)

See below for further reading on Kresladi:

BLA for Rocket’s LAD-I Gene Therapy Under Review by FDA Again

The FDA has accepted Rocket Pharmaceuticals’ resubmission of a biologics license application (BLA) for marnetegragene autotemcel (RP-L201, to be marketed as Kresladi), a genetically modified autologous hematopoietic stem cell therapy intended to treat leukocyte adhesion deficiency type 1 (LAD-I).

The FDA has set the Prescription Drug User Fee Act (PDUFA) action date for the BLA at March 28, 2026. Data from a global phase 1/2 clinical trial (NCT03812263) that showed a 100% overall survival rate in all enrolled patients for at least 1 year post treatment, and through the entire duration of follow-up, support the BLA. According to Rocket, this trial met all of its primary and secondary end points, with the incidence of significant infections having decreased substantially in comparison with levels before treatment and observed indications of skin lesion improvement and restored wound-healing capability. With regard to safety, Rocket noted that Kresladi was “well tolerated” and that there were no serious adverse events deemed related to treatment reported in the now completed study.

FDA Issues CRL for Rocket’s LAD-I Gene Therapy

The FDA has issued a complete response letter (CRL) to Rocket Pharmaceuticals’ marnetegragene autotemcel (RP-L201), to be marketed under the name Kresladi, for the potential treatment of leukocyte adhesion deficiency-I (LAD-I) due to a request for more Chemistry Manufacturing and Controls (CMC) information to complete its review.

Rocket Pharma stated that it had met with FDA officials and aligned on the “limited” information requested. The CRL marks the second time the FDA has requested more CMC information, after the agency extended its priority review for Kresladi for treating, pushing back the therapy’s Prescription Drug User Fee Act (PDUFA) date from March 31, 2024, to June 30 to allow additional time to review clarifying information submitted by Rocket in response to FDA information requests.