Rocket Pharmaceuticals Receives FDA RMAT Designation for Leukocyte Adhesion Deficiency-I

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The FDA granted the designation Regenerative Medicine Advanced Therapy (RMAT) to RP-L201. It is a therapy to treat Leukocyte Adhesion Deficiency-I (LAD-I). UCLA, University College London, and Hospital Infantil Universitario Niño Jesus are conducting this ongoing clinical study.

The FDA created the RMAT designation to advance the process and approval of regenerative medicine products, including cell and gene therapies. This decision results when a therapy is being researched to determine, with clinical data, that a therapy can help with an unmet medical need.

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease.  Mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18 cause this disease. The CD18 protein aids leukocyte adhesion and extravasation in order to overcome infections.

Frequent life-threatening infections cause much misery for these children. Unfortunately, mortality is very high for these children with most not living beyond age 5.

This non-randomized, open-label Phase 1/2 trial of RP-L201 will assess the safety and efficacy of the gene therapy in pediatric patients with severe LAD-I. CD18 expression is an important endpoint in this trial. Pediatric patients had CD18 expression passing the 4-10% range linked to enhanced life expectancy. The following quote is from Dr. Kinnari Patel, Chief Operating Officer for Rocket Pharmaceuticals.  

  “Receiving RMAT designation and completing Phase 1/2 patient enrollment are important steps in advancing our RP-L201 LAD-I program as efficiently and responsibly as possible. We look forward to maximizing the opportunity for enhanced dialogue with the FDA as we work closely with the agency on potential registration, thanks to the RMAT designation. Importantly, completing Phase 1/2 patient enrollment against the backdrop of a global pandemic is a testament to our team, collaborators, and the patients participating in the trial. I am grateful to all of them for their unwavering commitment as we seek to address the life-threatening impact of LAD-I on the lives of many infants, young children, and their families. We look forward to sharing data from our LAD-I trial in the second.”

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