Editas Medicine (EDIT) has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301. This is an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for sickle cell disease.
The genome editing company plans to file an investigational new drug application (IND) for EDIT-301 by the end of 2020.
“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines” said Cynthia Collins, CEO of Editas. “We are making tremendous progress towards this vision with the continued development of EDIT-301, a potentially transformative medicine for the treatment of sickle cell disease, and we are pleased to receive Rare Pediatric Disease designation from the FDA for this program” she continued.
The FDA defines a rare pediatric disease as a serious or life-threating disease in which the serious or life-threatening disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the United States.
Indeed, there are an estimated 100,000 people in the United States currently living with sickle cell disease. Sickle cell disease is an inherited blood disorder where misshapen red blood cells block blood flow causing anemia, pain crises, organ failure, and early death.
Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product.
EDIT-301 is comprised of sickle patient CD34+ cells genetically modified to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease. (See EDIT stock analysis on TipRanks).
Shares in Editas are up 15% year-to-date, and the stock scores a cautiously optimistic Moderate Buy Street consensus. That’s with an average analyst price target of $45, indicating upside potential of just over 30% from current levels.
“Overall, we continue to believe the company is well positioned to ultimately take advantage of the disruptive potential of CRISPR, but we remain on the sidelines given a potential lack of near-term value-creating catalysts” commented JP Morgan analyst Cory Kasimov earlier this month. He has a $30 price target on EDIT.
A similar message comes from Oppenheimer’s Jay Olson, who is also staying sidelined on the stock. “We believe EDIT will make progress in LCA10 and USH2A with in-vivo editing as well as SCD (sickle cell disease), beta-thalassemia, and cell therapies in oncology. However, we view the shares as fairly valued at the current price” he wrote on August 7.
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