FDA: RMAT Designation to Vertex and CRISPR’s TDT Therapy CTX001

Massachusetts-based Vertex Pharmaceuticals (NASDAQ: VRTX) and Swiss CRISPR Therapeutics (NASDAQ: CRSP) have announced the designation of investigational CRISPR gene-edited hematopoietic stem cell therapy CTX001 as a regenerative medication advanced therapy (RMAT) and orphan drug for the treatment of transfusion-dependent beta thalassemia (TDT) by the United States Food and Drug Administration (FDA) and as an orphan drug for both TDT and sickle cell disease (SCD) by the European Medicines Agency.

CEO of CRISPR Therapeutics Samarth Kulkarni, PhD, stated, “RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.”

“The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients,” commented Bastiano Sanna, PhD., Vertex Pharmaceutical’s Executive Vice President and Chief of Cell and Genetic Therapies.

Individuals suffering from SCD and TDT experience deficiencies in the ability of red blood cells to carry and distribute oxygen, potentially leading to a lack of sufficient oxygen in many cells throughout the body. CTX001 provides relief by inducing the expression of high levels of fetal hemoglobin (HbF; hemoglobin F), “a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin,” in red blood cells.

According to Vertex Pharmaceuticals, “The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.”

CTX001 is the first therapy to result from collaboration between CRISPR Therapeutics and Vertex initiated in 2015. CRISPR Therapeutics and Vertex Pharmaceuticals are operating under co-development and co-commercialization agreements for CTX001 and “will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.”

Established under the 21st Century Cures Act, RMAT designation is intended to expedite the development and approval process for drugs with the potential to “treat, modify, reverse or cure a serious or life-threatening disease or condition” given that “preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition.”

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