Editas Medicine Receives FDA Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Beta Thalassemia
Editas Medicine, Inc. (Nasdaq: EDIT) received Rare Pediatric Disease designation from the FDA for EDIT-301, a gene-edited treatment for beta thalassemia, complementing its previous designation for sickle cell disease. This designation underscores the urgent need for new therapies for severe conditions affecting children. The company plans to initiate a Phase 1/2 study for EDIT-301 in 2022, potentially benefiting patients requiring lifelong transfusions. If approved, Editas could obtain a Priority Review Voucher, enhancing its future product review processes.
- FDA granted Rare Pediatric Disease designation for EDIT-301, indicating strong regulatory support.
- Potential eligibility for a Priority Review Voucher upon marketing approval, which could expedite future product reviews.
- Plans to initiate a Phase 1/2 clinical trial for EDIT-301 in 2022, focusing on transfusion-dependent beta thalassemia.
- Dependence on successful clinical trials for EDIT-301, with inherent uncertainties.
- Potential risks related to regulatory approvals and funding for ongoing development.
CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia. The FDA previously granted Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease.
“Beta thalassemia is a devastating disease that leads 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,” said James C. Mullen, Chairman, President, and Chief Executive Officer, Editas Medicine. “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.”
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect fewer than 200,000 people in the United States.
Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, if Editas receives marketing approval for EDIT-301 for beta thalassemia, the Company may be eligible to receive a Priority Review Voucher (PRV) from the FDA that can be redeemed to receive priority review of a subsequent marketing application for a different product, or the PRV may be sold or transferred.
EDIT-301 is being investigated in a clinical study in patients with severe SCD (RUBY trial, NCT04853576). Editas expects to initiate a Phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta thalassemia in 2022.
About Beta Thalassemia
Beta thalassemia is a common autosomal recessive disorder with an estimated annual incidence rate of 1 in 100,000 worldwide for symptomatic individuals. Beta thalassemia mutations reduce or abrogate beta globin expression. Insufficient beta globin production leads to ineffective red blood cell production, chronic hemolytic anemia due to the destruction of red blood cells, and compensatory extramedullary hematopoiesis (creation of blood cells). Based on clinical severity and transfusion requirements, beta thalassemia can be classified into non-transfusion-dependent (NTDT) and transfusion-dependent beta thalassemia (TDT). TDT is the most severe form of beta thalassemia, and patients require lifelong regular red blood cell transfusions to prevent organ failure and death. Chronic red blood cell transfusions are complicated by iron overload leading to organ dysfunction and failure. Left untreated, the mortality rate among TDT patients is high, with a survival rate of only 15 percent at age five due to severe anemia.
About EDIT-301
EDIT-301 is an experimental cell therapy medicine under investigation for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). EDIT-301 consists of patient-derived CD34+ hematopoietic stem and progenitor cells edited at the gamma globin gene (HBG1 and HBG2) promoters, where naturally occurring fetal hemoglobin (HbF) inducing mutations reside, by a highly specific and efficient proprietary engineered AsCas12a nuclease. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin production, which has the potential to provide a one-time, durable treatment benefit for people living with severe sickle cell disease and transfusion-dependent beta thalassemia.
About Editas Medicine
As a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit www.editasmedicine.com.
Forward-Looking Statements
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words ‘‘anticipate,’’ ‘‘believe,’’ ‘‘continue,’’ ‘‘could,’’ ‘‘estimate,’’ ‘‘expect,’’ ‘‘intend,’’ ‘‘may,’’ ‘‘plan,’’ ‘‘potential,’’ ‘‘predict,’’ ‘‘project,’’ ‘‘target,’’ ‘‘should,’’ ‘‘would,’’ and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Company’s expectation to initiate a Phase 1/2 study of EDIT-301 in patients with TDT in 2022 and dosing the first patient in the TDT clinical trial this year. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release represent the Company’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. Except as required by law, the Company explicitly disclaims any obligation to update any forward-looking statements.
FAQ
What is the significance of the FDA granting Rare Pediatric Disease designation to EDIT-301?
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How does the Priority Review Voucher work for Editas if EDIT-301 is approved?
What is beta thalassemia?
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