The Biologics License Application (BLA) will be reviewed under the FDA’s Real-Time Oncology Review pilot program (RTOR program)
ElsaLys Biotech, a clinical stage company, owned by Mediolanum Farmaceutici Spa, announced today the U.S. Food and Drug Administration (FDA) agreement to start the LEUKOTAC® (inolimomab) submission process for a Biologics License Application (BLA) for the treatment of Steroid-Refractory acute graft-versus-host disease (aGvHD), grade II-IV adult patients.
The Biologics License Application (BLA) will be reviewed under the FDA’s Real-Time Oncology Review pilot program (RTOR program), an initiative of the FDA's Oncology Center of Excellence. The RTOR program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality by the FDA.
“This is an important milestone for ELSALYS BIOTECH as we are one step closer towards potentially bringing inolimomab to patients, responding to an increasing health need,” said Dr. Christine GUILLEN, CEO and co-founder of ElsaLys Biotech. “ I want to thank the team for their dedication in working to address the FDA’s findings over the past few months. We look forward to working with the FDA throughout its review process.”
Inolimomab has shown a robust and long-lasting response Rate in steroid-refractory acute graft versus host disease (SR-aGvHD) in a randomized multicentre controlled parallel-group Phase 3 study (INO-107 - EUDRACT 2007-005009-24). In addition, Inolimomab demonstrated a clear advantage in long-term survival compared to control group treated with ATG (Anti-Thymo Globulin). Affecting 30 to 55% of patients, aGvHD is the main complication of Hematopoietic Stem Cell Transplantation (HSCT) and is the major cause of morbidity and mortality in this setting. Around half of the patients with aGvHD do not respond to initial steroid treatment and are left with few therapeutic options.
Since December 24, 2019, the French National Agency for the Medicines and Health Products Safety (ANSM) granted a cohort ATU for inolimomab in France in this indication (also including pediatric patients).
“The increasing number of hematopoietic stem cell transplantation worldwide has triggered an increasing need for a large variety of new approved drugs addressing the acute Graft versus Host Disease and especially its Steroid-Resistant form that tend have a very poor outcome” said Dr. David LIENS, Chief Medical Officer, ElsaLys Biotech, concluding that “Robust clinical data including a positive safety profile, obtained from over 1400 treated individuals, positions inolimomab as a meaningful therapeutic alternative for THE treatment of steroid refractory aGvHD.”
In June 2020, FDA accepted the application for Leukotac under the RTOR pilot program which is a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible.
RTOR program is granted BY FDA to therapies that have the potential to demonstrate substantial improvements over available therapy.
This allows the FDA to review much of the data earlier, before the applicant formally submits the complete application.
Additional information about RTOR can be found at: https://www.fda.gov/about-fda/oncology-center-excellence/real-time-oncology-review-pilot-program.
The next step in the BLA Submission process to the FDA will be the Chemistry, Manufacturing and Controls (CMC) pre-BLA meeting in September 2020.