This morning, it was announced that the U.S. Food and Drug Administration (FDA) has approved the gene therapy Luxturna (voretigene neparvovec-rzyl) to treat children and adult patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
In October, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee met to review data presented by Spark Therapeutics and voted 16-0 in favor of the drug. Today’s regulatory action marks the second approval for a gene therapy in the United States. Kymriah was approved for the treatment of acute lymphoblastic leukemia (ALL) in August.
“Today’s approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases," said FDA Commissioner Scott Gottlieb, M.D. in the official FDA press release
. The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses.”
The drug was previously granted Priority Review and Breakthrough Therapy Designations, as well as an Orphan Drug Designation, and the approval was largely based on data from a Phase 3 study that was published in Lancet
in August, which exhibited the drug’s effectiveness. In that study, 20 patients were administered the gene therapy, and at 1 year, mean bilateral multi-luminance mobility testing (MLMT) change score was 1.8 (standard deviation = 1.1) light levels in the gene therapy group versus 0.2 (standard deviation = 1.0) in the control group (P = .0013).
While the 3-year data is important for demonstrating the durability and effect of the drug, the durability curve at 3 years for patients who have received Luxturna looks nearly identical to the 1- and 2-year time points. To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.
The approval comes almost one month ahead of the scheduled Prescription Drug User Fee Act (PDUFA) date of January 12, 2018. “I’ve never heard of any drug being approved before it’s PDUFA date,” said Stephen R. Russell, M.D., of the University of Iowa, one of the lead researchers on the study in an exclusive interview
with Rare Disease Report
in November, “but I guess it could happen.”
“The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.”
It is suggested by the FDA that Luxturna be administered only to patients who have viable retinal cells as determined by the treating physicians, and treatment with the drug must be done separately in each eye on separate days, with at least 6 days between surgical procedures. The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.
Spark Therapeutics is receiving a Rare Pediatric Disease Priority Review Voucher, the 13th
distributed by the FDA since the initiation of the program.
For more from the FDA, including applications, designations and approvals, follow Rare Disease Report