The US Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee have completed its meeting for CRISPR Therapeutics and Vertex Pharmaceuticals’ exagamglogene autotemcel (exa-cel) to treat severe sickle cell disease (SCD).
The potential therapy is the first to emerge from CRISPR’s and Vertex’s strategic partnership, which was expanded in 2021.
Affecting more than 20 million people worldwide, SCD is a group of inherited red blood cell disorders which cause red blood cells to become hard and sticky and appear in a c-shaped form known as ‘sickle’.
The lifelong illness can lead to serious health problems, including strokes, eye problems, infections and episodes of pain.
Exa-cel will be used to treat people aged 12 and older with SCD and recurrent vaso-occlusive crises, when sickled red blood cells block blood flow and deprive tissues of oxygen.
The FDA previously granted priority review for exa-cel to treat people with SCD with a prescription drug user fee action date of 8 December 2023.
If approved, the candidate will be the first genetic therapy to be available to approximately 20,000 people in the US with severe SCD.
CRISPR and Vertex entered into a strategic research collaboration in 2015 to discover and develop potential new treatments for underlying genetic causes of human disease, focusing on the use of a gene-editing technology involving ribonucleic acid and CRISPR-associated protein 9 (Cas9).
In June, CRISPR and Vertex announced positive results from two pivotal trials, CLIMB-111 and CLIMB-121, which showed that exa-cel in patients with transfusion-dependant beta-thalassaemia (TDT) for severe SCD met its primary and key secondary endpoints.
TDT, a serious form of beta-thalassaemia – an inherited blood disorder that prevents the body from producing enough beta-globin – affects the body’s haemoglobin production.
“The updated results from both the TDT and SCD trials… bring promise of an autologous CRISPR/Cas9 gene-edited cell therapy one-step closer to patients who are waiting,” said Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex.
Results from the trials were presented at the 2023 Annual European Hematology Association Congress in June.