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US FDA puts hold on CRISPR, Vertex's application for sickle cell disease drug

The U.S. Food and Drug Administration placed a clinical hold on CRISPR Therapeutics AG and Vertex Pharmaceuticals Inc.'s investigational new drug application for CTX001 to treat sickle cell disease.

Sickle-cell disease is a group of blood disorders normally inherited from a person's parents.

The clinical hold will remain till the resolution of certain questions that will be provided by the agency as part of its review of the application, which was submitted in April to support the planned initiation of a phase 1/2 trial in the U.S. in adult patients with sickle cell disease.

CRISPR and Vertex, which are jointly developing the drug under a co-development and co-commercialization agreement, expect to obtain additional information on the FDA's questions and plan to work with the regulator toward a resolution.

The companies said the planned initiation of a phase 1/2 trial of the treatment in Europe in adult patients with transfusion-dependent beta-thalassemia is unchanged and expected to be initiated in the second half of 2018.