A Spark Points the Way Forward on Gene Therapy

Today (October 12, 2017) FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee unanimously recommended approval of Spark Therapeutics, Inc.’s Biologics License Application (BLA 125610) for Voretigene Neparvovec (also called LUXTURNA), a gene therapy for the treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy (i.e., an inherited retinal dystrophy that always progresses to blindness). This is the first true gene therapy to come before an FDA advisory committee.  Today’s action boosts not only the hopes of those with this specific genetic disorder, but also sparks hope among all those with incurable genetic diseases.

“Lessons Learned” include:

• A single pivotal efficacy study of modest scope (n=31) may be sufficient.
• Dose optimization and drug safety assessments, even for highly innovative gene therapy, need not be necessarily more extensive than those for other similarly rare diseases.
• Videographic evidence of subjects negotiating a dimly-lit maze shows sponsor and FDA commitments to preference for a patient-centric primary endpoint measure.
• This measurement of a critical daily patient function is even more noteworthy here in this clinical area that has very well established quantitative measures (e.g., visual acuity and visual field which were secondary endpoints).

Global Observation:

The Office of Tissues and Advanced Therapies within the Center for Biologics Evaluation and Research at FDA exhibits reasonable and appropriate “flexibility” in its review of this first true gene therapy seeking FDA registration.

Today’s action may be the Spark that illuminates the way forward for all those with incurable genetic disease.