In what is the first in the field of gene therapy, Spark Therapeutics’ Luxturna has been granted Food and Drug Administration (FDA) approval.
Shares of the stock quickly soaed almost 7% before retracing and closing down 0.2% on Tuesday.
According to FDA Commissioner Scott Gottlieb, the approval marks another first in gene therapy. The approval for Luxturna is also the third gene therapy that the FDA has approved.
The company’s treatment is used for a rare form of inherited vision loss called Leber congenital amaurosis. The condition is rare and is a genetic retinal disease.
Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene where the person has a mutated RPE65 gene.
This is the gene that is important in making an enzyme that is needed to have normal vision.
FDA Commissioner Scott Gottlieb remarked, “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.”
Disclaimer: We have no position in Spark Therapeutics, Inc. (NASDAQ: ONCE) and have not been compensated for this article.