Casgevy is a genetically modified autologous CD34+ cell-enriched population that contains human hematopoietic stem and progenitor cells, edited using CRISPR/Cas9 technology, developed by Vertex Pharmaceuticals and CRISPR Therapeutics
Vertex Corporate Headquarters - Boston Seaport. (Credit: Vertex Pharmaceuticals Inc.)
UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
Casgevy is a genetically modified autologous CD34+ cell-enriched population with human hematopoietic stem and progenitor cells, edited using CRISPR/Cas9 technology.
Previously known as CTX-001, Casgevy is the first treatment to emerge from a research collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics, that started in 2015.
The drug is authorised to treat SCD in eligible patients, aged 12 years and above, with recurrent vaso-occlusive crises (VOCs) for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related hematopoietic stem cell donor is not available.
Also, it is indicated for the treatment of TBT in patients aged 12 years and above, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.
Casgevy has been evaluated in two global clinical trials in SCD and TDT, which met their respective primary outcome of becoming free from severe VOCs for at least 12 months.
It showed a safety profile that is consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant, in the ongoing studies on SCD and TDT patients.
Vertex president and CEO Reshma Kewalramani said: “Today is a historic day in science and medicine: this authorization of CASGEVY in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world.”
CRISPR Therapeutics chairman and CEO Samarth Kulkarni said: “I hope this represents the first of many applications of this Nobel Prize-winning technology to benefit eligible patients with serious diseases.”
CLIMB-111 and CLIMB-121 studies principal investigator Josu de la Fuente said: “This authorization offers a new option for eligible patients who are waiting for innovative therapies, and I look forward to patients having access to this therapy as quickly as possible.”
Vertex has secured an Innovation Passport for Casgevy, under MHRA’s Innovative Licensing and Access Pathway (ILAP) and is working closely with national health authorities.
The drug is also under review by the European Medicines Agency (EMA), the Saudi Food and Drug Authority, and the US Food and Drug Administration (FDA).
The US health regulator granted Priority Review for SCD and Standard Review for TDT, with Prescription Drug User Fee Act (PDUFA) dates of 8 December 2023, and 30 March 2024.