The one-time treatment helped relieve symptoms of disease and could free patients from the need for bone marrow transplants or regular blood transfusions, Beach said, adding that the drug hopefully offers a permanent fix for the condition.
The MHRA said it identified no significant safety concerns during the trials and will continue to closely monitor Casgevy’s safety after approval.
Vertex CEO and President Reshma Kewalramanit celebrated Casgevy’s approval as “a historic day in science and medicine” and Samarth Kulkarni, CEO and Chairman of Crispr Therapeutics, said it will hopefully mark “the first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases.” The two companies are hoping for similarly positive decisions from the MHRA’s counterparts in the Europe Union and the U.S., which are in the process of evaluating Casgevy, also known as exa-cel. The Food and Drug Administration is expected to make a decision in early December and has a deadline of December 8. The agency appears poised to follow the MHRA and approve the treatment, with its advisors confident of the drug’s efficacy and benefit but wary of theoretical unintended consequences of genetic modifications.
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