Clinical Trials:
Leukocyte Adhesion Deficiency-I (LAD)
RP-L201 is being developed for severe LAD-I, a rare genetic immune disorder that predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant (HSCT).
The Biologics License Application (BLA) has been accepted with Priority Review by the U.S. Food and Drug Administration (FDA), based on the positive efficacy and safety data from the Phase 1/2 study of RP-L201 for severe LAD-I. The FDA’s review of the BLA is ongoing.
Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com.
For more details about the trial, visit clinicaltrials.gov.
To read our Expanded Access statement, click here.