A panel of experts said on Tuesday that a groundbreaking treatment for sickle cell disease was safe enough for clinical use, setting the stage for likely federal approval by Dec. 8 of a powerful potential cure for an illness that afflicts more than 100,000 Americans.
The Food and Drug Administration had previously found that the treatment, known as exa-cel and jointly developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Switzerland, was effective. The panel’s conclusion on Tuesday about exa-cel’s safety sends it to the F.D.A. for a decision on greenlighting it for broad patient use.
Exa-cel frees patients from the debilitating and painful effects of this chronic, deadly disease. If approved, the Vertex product would be the first medicine to treat a genetic disease with the CRISPR gene-editing technique.
It could also be the first of a series of new options to cure the excruciating illness. By Dec. 20, the F.D.A. will decide on a second potential cure for sickle cell, a gene therapy devised by the company Bluebird Bio of Somerville, Mass.
Sickle cell disease is caused by a gene mutation that makes blood cells misshapen, so that they resemble sickles or crescents. It affects millions of people worldwide, most of whom have African ancestry. The misshapen cells get stuck in blood vessels, causing strokes, organ damage and episodes of agonizing pain as muscles are starved of oxygen.
Sickle cell’s toll starts early in life. Evelyn Islam of Milwaukee, now 8, had 22 blood transfusions and had to have her spleen removed before she was 3. “Gene therapy is our last hope for a cure,” said her mother, Melissa Nicole Allen.
