Editas, VBL and Pharming Score Regulatory Wins On Development Journey

Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

Editas Medicine snagged Rare Pediatric Disease designation for its experimental beta thalassemia gene therapy, while VBL received Fast Track designation for an ofra-vec combination in ovarian cancer.

Sarah Silbiger/Getty Images

Editas Medicine snagged Rare Pediatric Disease designation for its experimental beta thalassemia gene therapy EDIT-301. The designation, awarded by the U.S. Food and Drug Administration, comes ahead of the initiation of a Phase I/II study later this year.

The designation for beta thalassemia follows a previous Rare Pediatric Disease designation for EDIT-301 for the treatment of sickle cell disease. EDIT-301 is designed to edit the HBG1/2 promoter to disrupt the binding site of BCL11a and ameliorate disease symptoms.

James C. Mullen, the outgoing chief executive officer of Editas, expressed excitement over the newly-awarded designation for beta thalassemia, a blood disease that can lead to severe anemia, organ failure and premature death.

“Receiving Rare Pediatric Disease designation for EDIT-301 for beta thalassemia highlights the dire need for new treatment options. EDIT-301 is a potentially transformative medicine for patients living with beta thalassemia, and we look forward to dosing the first patient in our clinical trial this year,” Mullen said in a statement.

The designation is awarded to investigational therapeutics developed for rare pediatric diseases that primarily affect individuals aged from birth to 18 years. The rare designation is used by the FDA for disease indications that affect fewer than 200,000 people in the United States.

If EDIT-301 wins approval, Editas could be eligible to receive a Priority Review Voucher that can be sold to another company or used to advance the potential approval of another asset.

Editas wasn’t the only company to secure a designation from the FDA. Israel-based VBL Therapeutics received Fast Track designation from the FDA for ofra-vec (ofranergene obadenovec or VB-111) in combination with paclitaxel for the treatment of platinum-resistant ovarian cancer.

Ofra-vec is VBL Therapeutics’ investigational anti-cancer, gene-based agent in development to treat a wide range of solid tumors, including ovarian cancer. Ofra-vec is in Phase III development for ovarian cancer. The developmental drug will be assessed in the Phase III OVAL study that recently received the green light to continue from the Independent Data Safety Monitoring Committee after a review of unblinded data from 370 randomized patients.

VBL CEO Dror Harats said the newly-received designation can facilitate the regulatory process for ofra-vec should it meet its clinical endpoints of progression-free survival and overall survival. The PFS readout of the OVAL study is expected in the third quarter of the year, Harats said. A top-line readout of the OS primary endpoint is anticipated in 2023.

“We believe that, if positive, this will support a Biologics License Application submission to the FDA,” Harats said in a brief statement.

Across the ocean, Netherlands-based Pharming Group scored a positive decision from the UK’s Medicines and Healthcare Products Regulatory Agency for a Pediatric Investigation Plan (PIP) submission for the company’s phosphoinositide 3-kinase delta (PI3Kδ) inhibitor leniolisib, which is being developed for the treatment of the rare disease, activated phosphoinositide 3-kinase delta syndrome (APDS) in patients ages 1 to 18. Additionally, the U.K.’s MHRA granted Promising Innovative Medicine (PIM) designation to leniolisib for the treatment of APDS.

For the U.K. regulatory agency, a PIP is awarded to ensure the necessary data is provided that will support marketing authorization. The leniolisib PIP includes two planned global clinical trials in pediatric patients with APDS. The first such data is in children ages 4-11 years and the second in children ages 1-6 years. The company plans to initiate recruitment for this pediatric program for leniolisib during the second half of 2022.