FDA Grants Orchard RMAT Designation for Wiskott-Aldrich Syndrome Gene Therapy

The U.S. Food and Drug Administration granted Orchard Therapeutics Regenerative Medicine Advanced Therapy designation for OTL-103, the company’s gene therapy for the treatment of Wiskott-Aldrich Syndrome, a life-threatening autoimmune disorder.

Wiskott-Aldrich Syndrome (WAS) manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. It is characterized by eczema and an abnormal platelet number and function. Without treatment, the median survival for WAS patients is 14 years of age and treatment with stem cell transplant carries significant risk of mortality and morbidities.

OTL-103 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan for the treatment of WAS, which Orchard acquired from GSK in April 2018.

Data from an interim analysis of a registrational trial in patients with severe WAS published in May 2019 in The Lancet Haematology describes recovery of the immunological and platelet abnormalities associated with WAS, with a consequent significant reduction in the major complications of the disease.  

Following treatment with OTL-103 gene therapy, patients with follow-up ranging from 0.5 to 5.6 years showed a decrease in the frequency of severe infections. In addition, severe bleeding episodes were eliminated, and moderate bleeding episodes were greatly reduced.

The FDA grants Regenerative Medicine Advanced Therapy (RMAT) designation to experimental therapies intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, when preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for such disease or condition.

The designation provides for expedited drug development and review processes for promising pipeline products, including gene therapies. It gives companies access to intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA senior management on a range of development and review issues.  

The company said it remains on track to file for regulatory approval for its WAS gene therapy program in the United States and Europe in 2021.

“This is good news for the WAS community both in the U.S. and worldwide, since it could speed patients’ access to a potentially life-saving treatment for this serious condition,” said Alessandro Aiuti, professor of Pediatrics at the Vita-Salute San Raffaele University and vice director of SR-Tiget.

Photo: Alessandro Aiuti, professor of Pediatrics at the Vita-Salute San Raffaele University and vice director of SR-Tiget

Author: Rare Daily Staff