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FDA Approves Luxturna One Month Before PDUFA Date

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.”

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