BY LAURIE MCGINLEY
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 hemophilia, sickle-cell anemia and an array of other genetic diseases. Yet the products, should they reach patients, are likely to carry stratospheric prices — a prospect already worrying consumer advocates and economists.
Philadelphia-based Spark Therapeutics, which makes the childhood-blindness treatment, said it will not announce the price until January. Analysts speculate it could be as much as $1 million for both eyes. Continue Reading →