FDA
21st November 2017

FDA Advisory Committee Unanimously Recommends Approval of LUXTURNA for Patients with Biallelic RPE65-mediated Inherited Retinal Disease

On October 12th, the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met to discuss and make recommendations.

FDA Advisory Committee Unanimously Recommends Approval of LUXTURNA for Patients with Biallelic RPE65-mediated Inherited Retinal Disease

On October 12th, the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met to discuss and make recommendations on the safety and effectiveness of the Biologics License Application (BLA 125610) for LUXTURNA™ (voretigene neparvovec), submitted by Spark Therapeutics, Inc. The proposed indication is treatment of patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy.

LUXTURNA is an adeno-associated virus 2 (AAV2) vector containing human RPE65 complementary DNA (cDNA). It is a one-time therapy that is injected subretinally under general anesthesia. The BLA for LUXTURNA is supported by a randomized controlled open-label study evaluating LUXTURNA in participants with RPE65-mediated inherited retinal dystrophy. The clinical program for LUXTURNA includes patient data that show efficacy for up to four years on endpoints including bilateral multi-luminance mobility test (MLMT) score change and full-field light sensitivity threshold (FST) testing. LUXTURNA demonstrated efficacy and safety in a phase 3 trial which included patients ages 4-44 with sufficient viable retinal cells from vision loss due to bialleic RPE65 mutations. The experimental group reported statistically significant and clinically meaningful improvements versus control in the mean bilateral multi-luminance mobility testing (MLMT) score at 1 year.

The FDA’s opinion was that LUXTURNA improved sight using the light levels measured for the primary endpoint, but fell short of curing retinal dystrophy triggered by genetic RPE65 mutations. Improvement in vision was also limited by the number of viable retinal cells patients had left at the time of treatment. First, the committee was asked whether a 2-light level improvement in MLMT is clinically meaningful in patients with vision loss due to confirmed biallelic RPE65 mutation-associated retinal dystrophy.  The committee members were also asked questions about the durability of this eye therapy, the possibility that patients will need multiple treatments in order to preserve vision gains, the right age to use it and the use of a novel endpoint for the primary goal when standard visual acuity achievements were not statistically significant. Overall, there were 3 discussion questions and 1 voting question in this meeting.

The vote was 16 to 0 favoring the benefit-risk profile of LUXTURNA. There currently are no pharmacologic treatment options for people living with RPE65-mediated IRD, who in most cases progress to complete blindness. In addition, no serious adverse events (SAEs) associated with LUXTURNA or deleterious immune responses were observed during the study. The Agency will take this vote into consideration when reviewing the BLA for LUXTURNA, which is under Priority Review. The FDA is not obligated to follow the committee’s recommendation, but normally does so.  An FDA decision on the voretigene neparvovec BLA is expected by mid-January 2018.

ISS has almost two decades of experience in developing regulatory strategies including support for FDA Advisory Committee meetings. We are involved in more FDA AdComms per year than even the largest pharmaceutical companies. For more information on how ISS can help you prepare for your next meeting, contact info@innovativescience.net

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