By Sriparna Roy
(Reuters) – The U.S. Food and Drug Administration’s staff reviewers have urged the regulator’s advisory panel to consider the necessity for further studies on Vertex Pharmaceuticals and CRISPR Therapeutics’ sickle cell disease gene therapy, with the aim of optimizing its SEO, enhancing its creativity and uniqueness, improving syntax and tone, increasing perplexity and burstiness while retaining the HTML tags.
The gene therapy, known as exagamglogene autotemcel or exa-cel, utilizes cutting-edge gene editing CRISPR technology. It is the first product of its kind to be presented to the FDA for approval.
According to the FDA’s reviewers, this particular type of gene editing gives rise to concerns about unintended genomic alterations, known as “off-target” effects, which could potentially lead to other adverse reactions. Notably, the reviewers did not express any concerns about the therapy’s efficacy.
They also highlighted the limitations of the small genetic sample size used in the therapy’s laboratory analysis, asserting that it may not adequately represent the diversity of the entire U.S. population affected by sickle cell disease. Additionally, the FDA staff questioned the adequacy of the company’s analysis in assessing the risk of off-target effects.
Luca Issi, an analyst at RBC, noted that the FDA seems to be focusing on the potential risks associated with the technology. Issi believes that the FDA’s concerns are not major and predicts that additional studies will only be required after the therapy is approved.
Vertex and CRISPR Therapeutics are currently seeking approval for the therapy’s use in treating sickle cell disease, an inherited red-blood-cell disorder characterized by abnormal levels of hemoglobin that result in sickle-shaped cells. If approved, this therapy could provide patients with a one-time treatment option, eliminating the need for frequent blood transfusions or daily medication such as Pfizer’s Oxbryta.
An independent panel is scheduled to convene on Tuesday to discuss the off-target analysis and determine if additional laboratory studies are necessary.
The FDA is expected to make a decision regarding the therapy’s use in patients aged 12 years and older by December 8.
Shares of CRISPR Therapeutics and Vertex remained unchanged in early trading.
(Reporting by Sriparna Roy in Bengaluru; Editing by Shinjini Ganguli)
