RARE Daily

FDA Grants RMAT Designation to Rocket’s Gene Therapy for Rare Pediatric Disease LAD-I

March 9, 2021

Rare Daily Staff

 The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy designation to Rocket Pharmaceuticals’ RP-L201, its experimental gene therapy for the treatment of the rare pediatric disorder Leukocyte Adhesion Deficiency-I.

Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75 percent prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Regenerative Medicine Advanced Therapy (RMAT) designation was granted based on encouraging preliminary safety and efficacy data from the ongoing phase 1/2 clinical trial of RP-L201, an experimental lentiviral-based gene therapy. The ongoing, non-randomized, open-label study is designed to evaluate the safety and efficacy of the gene therapy in pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2 percent. Data from the study demonstrated evidence of safety and efficacy in three pediatric patients with severe LAD-I. These patients have shown sustained CD18 expression exceeding the 4-10 percent threshold associated with survival into adulthood and similarly encouraging peripheral blood vector copy numbers. RP-L201 was well tolerated with no drug product safety issues reported with infusion or post-treatment.

“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,” said Kinnari Patel, president and chief operating officer of Rocket. “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.”

RMAT designation was established to help expedite the development and approval of regenerative medicine products, including cell and gene therapies. The designation is granted to an investigational product that intends to treat, modify, reverse or cure a serious or life-threatening disease or condition, and is supported by preliminary clinical evidence demonstrating its potential to address an unmet medical need. RMAT designation allows companies to work closely with the FDA on a program’s development and includes all the benefits of the FDA’s Fast Track and Breakthrough Therapy designations. Rocket also holds Rare Pediatric (U.S.), Orphan Drug (U.S./EU), and Advanced Therapy Medicinal Product (EU) designations for the RP-L201 program.

RP-L201 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The lentiviral vector was developed in collaboration between UCL and CIEMAT.


Stay Connected

Sign up for updates straight to your inbox.