Spark Therapeutics, a Philadelphia-based drug development company, recently announced that the Food and Drugs Administration had received its request for a biologics license and offered it priority review for its principal drug candidate that will be used in the treatment of a rare blindness that is hereditary. The new drug will become the first gene-based therapy for curing a genetic condition in the United States if it is approved. The treatment is called voretigene neparvovec, and it works by steaming genes to an individual’s eye retina. The FDA gave the therapy a priority review because it focuses on a medical condition where there is no therapy has been developed so far.
The new Spark treatment might take about six months to be approved. The company was created at the Children’s Hospital of Philadelphia and has conducted research for decades under the leadership of Katherine A. High who is its co-founder, chief scientific officer, and president. The CEO of Spark, Jeffrey D. Marrazzo, said that it is an exciting moment for the medical community. The FDA recently examined an experimental T-cell immune therapy that is being created by the University of Pennsylvania and Novartis to cure acute lymphocytic leukemia. The first studies of the CAR-T cell technology were carried out at the Children’s Hospital of Philadelphia.
The company believes that the FDA might consider creating a team of medical experts to review its new treatment if it fails to use data from three clinical experiments that involved 41 patients. In phase 3 of the study, 27 out of 29 individuals who had been enrolled improved their vision. The gene therapy does not have any serious side effects, but two cases were reported to have been caused by the surgery, which involves being injected into the eye. One person got a bacterial infection while the other lost sharpness of vision after the injection.
Spark Therapeutics has been monitoring the patients who are in the earlier phase 1 of the trial for the past four years, and they have been improving their vision. Approximately 3,500 patients in the United States and five top European markets are affected by the condition. Fifty percent of them live in the U.S. The treatment that has been developed by Spark involves injecting particles that resemble the normal functioning gene the back of the patient’s eyes. The firm hopes that voretigene neparvovec will be a one-time treatment, but its price has not yet been set.