FDA panel says Vertex/CRISPR to assess safety risks of gene therapy in
follow-up study
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[November 01, 2023]
(Reuters) - A panel of advisers to the U.S. health regulator said
on Tuesday Vertex Pharmaceuticals and CRISPR Therapeutics could assess
potential safety risks of their sickle cell disease gene therapy after
approval.
If the therapy is approved, Vertex has proposed a 15-year follow up of
patients to evaluate the safety outcomes of the therapy.
The inherited red-blood-cell disorder causes the cells to become
sickle-shaped due to abnormal levels of hemoglobin in the body. A vaso-occlusive
crisis occurs when sickled red blood cells block blood flow to the point
that tissues become deprived of oxygen and causes pain.
The panel members said the 15-year follow up will help generate data
from real-time monitoring of the therapy, which uses the new gene
editing CRISPR technology.
Staff reviewers to the U.S. Food and Drug Administration said the new
type of technology raised concerns about the "off target", or unintended
genomic alterations that can potentially cause other side effects, but
did not raise any concerns about the therapy's efficacy.
The therapy, exagamglogene autotemcel or exa-cel, met the main goal of a
late-stage study. Patients who were treated with the therapy were free
from severe vaso-occlusive crisis for 12 months from the infusion of
exa-cel.
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A sign hangs in front of the world headquarters of Vertex
Pharmaceuticals in Boston, Massachusetts, U.S., October 23, 2019.
REUTERS/Brian Snyder/File Photo
"With regards to off target analysis
we want to be careful to not let the perfect be the enemy of the
good," said Dr. Scot Wolfe, professor at the department of
molecular, cell and cancer biology at UMass Chan Medical School.
Analysts have been optimistic the therapy, which is a
first-of-its-kind product to reach the FDA for review, will win the
health regulator's nod by Dec. 8.
"It was made pretty clear that any theoretical off-target editing
concerns do not outweigh the benefits of drug approval," said Salim
Syed, analyst at Mizuho.
(Reporting by Sriparna Roy in Bengaluru; Editing by Krishna Chandra
Eluri)
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