In a historic move, the Food and Drug Administration on Tuesday approved a pioneering gene therapy for a rare form of childhood blindness, the first such treatment cleared in the United States for an inherited disease.
The approval signals a new era for gene therapy, a field that struggled for decades to overcome devastating setbacks but now is pushing forward in an effort to develop treatments for haemophilia, sickle-cell anaemia, and an array of other genetic diseases.
Earlier this year, the FDA approved two other treatments it considers gene therapy. The eye remedy cleared Tuesday represents a classic goal of gene therapy – to directly treat disorders that are caused by a faulty gene.
“Today’s approval marks another first in the field of gene therapy – both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss,” said FDA Commissioner Scott Gottlieb in a statement.
The gene therapy field was derailed years ago after a series of high-profile failures, including the 1999 death of teenager Jesse Gelsinger who was participating in a University of Pennsylvania clinical trial for a rare liver disorder.
The two gene therapies that the FDA approved earlier this year were Novartis’ Kymriah, which is for childhood leukaemia that doesn’t respond to other treatments and Gilead Sciences’ Yescarta, which is for hard-to-treat lymphoma in adults.