US-FDA approves Vertex Casgevy to treat transfusion-dependent beta thalassemia

Vertex Pharmaceuticals Incorporated announced that the US Food and Drug Administration (FDA) has approved Casgevy (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy, for the treatment of transfusion-dependent beta thalassemia (TDT) in patients 12 years and older.

“On the heels of the historic FDA approval of Casgevy for sickle cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, M.D., chief executive officer and president of Vertex. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing Casgevy to eligible patients who are waiting.”

The administration of Casgevy requires experience in stem cell transplantation; therefore, Vertex is engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers (ATCs) throughout the US to offer Casgevy to patients. All nine ATCs activated in the US are able to offer Casgevy to eligible patients with TDT and sickle cell disease (SCD).

TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and the lifetime health care costs in the US of managing TDT are estimated between $5 and $5.7 million. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anaemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In the US, the median age of death for patients living with TDT is 37 years. Stem cell transplant from a matched donor is a curative option but is only available to a small fraction of people living with TDT because of the lack of available donors.

Casgevy is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal haemoglobin (HbF; haemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying haemoglobin that is naturally present during foetal development, which then switches to the adult form of haemoglobin after birth. Casgevy has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.

Casgevy is approved for certain indications in multiple jurisdictions for eligible patients.

Vertex Connects is a program for eligible patients in the US who have been prescribed Casgevy. Through Vertex Connects, Care Managers are available to provide educational resources, communications and support to navigate the treatment journey, and additional assistance is available for eligible patients.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.

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