CRISPR Therapeutics, in combination with Vertex Pharmaceuticals, has recently announced that the European Medicines Agency (EMA) has granted the Priority Medicines (PRIME) designation for their sickle cell disease (SCD) treatment, CTX001.
About Sickle Cell Disease (SCD)
Sickle cell disease (SCD) includes a group of disorders that are characterized by malformed red blood cells that look like sickles. The most common and severe form of this disease is sickle cell anemia. In sickle cell disease, deformed cells cause blockages and restricted blood flow as they get caught along the walls of blood vessels. This disease is caused by a mutation in the gene responsible for producing hemoglobin, which carries oxygen throughout the body. This gene is inherited in an autosomal recessive pattern, meaning both parents must pass down a copy to their child. Sickle cell disease is most common in people of African and Hispanic descent.
Symptoms of sickle cell disease include pain crisis, swelling of the hands and feet, and symptoms associated with anemia like fatigue, jaundice, and delayed growth. Adults tend to constantly feel the effects of this disease, but children usually only experience them during pain crisis. Regardless of age, damage usually occurs to the organs that are affected by the blocked blood flow. The most commonly damaged organs are the brain, eyes, spleen, liver, kidneys, lungs, heart, skin, joints, and bones. There is no cure for sickle cell disease. While some people qualify for bone marrow and blood transplants, not everyone is eligible for this procedure. Other forms of treatment are symptomatic and meant to prolong life.
About the PRIME Designation
PRIME is a regulatory mechanism that aims to help the progression of innovative therapies. The EMA grants this designation to promising therapies so that they can reach patients faster. They optimize development plans, along with speeding up the evaluation process.
CTX001 for Sickle Cell Disease
CTX001 is a CRISPR/Cas9 gene therapy that is being developed to treat severe sickle cell disease and transfusion-dependent beta thalassemia. It is intended to engineer the stem cells to produce more fetal hemoglobin (HbF). Higher levels of HbF alleviate the symptoms of the disease and lower the need for transfusions.
It has already been granted the Fast Track, Regenerative Medicine Advanced Therapy, and Orphan Dug designations from the FDA in the United States, along with the Orphan Drug designation from the European Commission.
Further decisions made by regulatory agencies will be based on an ongoing CLIMB-121 trial, which is evaluating the efficacy and safety of CTX-001 in SCD patients. Up to 45 patients will participate and be assessed for two years. This trial is running in tandem with the CLIMB-111 trial, which is a Phase 1/2 that will evaluate the safety and efficacy of the treatment in patients with beta thalassemia.
Hopefully these trials produce positive results so that CTX-001 can reach patients and better lives. Read more about this treatment here.