FDA approves Gene Therapy for rare form of blindness

FDA approves Gene Therapy for rare form of blindness

The U.S. Food and Drug Administration has approved the first ever gene therapy for a rare form of inherited blindness. The approval marks a major milestone for genetic medicine. Spark Therapeutics has received FDA approval for gene therapy in which a corrective gene is directly given to patients. The injection will be marketed under brand name Luxturna. The treatment has been approved for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss.

Patients with rare condition generally start losing vision before 18 and in majority of cases, the condition results in total blindness. The company said that the patients who benefitted from the genetic therapy, have witnessed lasting benefits. The company tracked the patients for nearly four years.

Market analysts estimate the price of treatment could be as high as $1 million. Spark Therapeutics informed that the company will reveal the cost of treatment next month. Pharmaceutical companies have been working on gene therapy but majority of approved treatments have very high cost. Novartis' Kymriah costs $475,000 for a one-time infusion of genetically enhanced cells. Gilead Sciences offers Yescarta for $373,000 per treatment.

Talking about approval, FDA Commissioner Scott Gottlieb, M.D. said, “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening. Next year, we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters — including new clinical measures — for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.”

Spark CEO Jeffrey Marrazzo said, "All the data we have today suggests it's long-lasting, if not lifelong."

The safety and efficacy of Luxturna were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subject’s ability to navigate an obstacle course at various light levels. The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.

Spark Therapeutics stock touched high of $50.74 and was trading slightly lower during the afternoon session. The stock price of Philadelphia, Pennsylvania-based company has declined from its recent high touched in October 2017.