Vertex and CRISPR Therapeutics Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency to CTX001 for Transfusion-Dependent Beta Thalassemia
BOSTON and CAMBRIDGE, Mass. and ZUG, Switzerland, April 26, 2021 (GLOBE NEWSWIRE) -- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)
Therapeutics (Nasdaq: CRSP) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo
CRISPR/Cas9 gene-edited therapy for the treatment of transfusion-dependent beta thalassemia (TDT). CTX001 was previously granted PRIME designation for the treatment of sickle cell disease (SCD) in
PRIME is a regulatory mechanism that provides early and proactive support to developers of promising medicines to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical data from CRISPR and Vertex’s ongoing Phase 1/2 trial of CTX001 in patients with TDT.
Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from EMA for both TDT and SCD.
CTX001 is being investigated in two ongoing clinical trials as a potential one-time therapy for patients suffering from TDT and severe SCD.
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.
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