Spark Gene Therapy Improves Vision, at Least in Short Term-FDA - (The New York Times via NewsPoints Desk)

  • FDA advisors disclosed in documents released ahead of an advisory panel meeting that Spark Therapeutics' experimental gene therapy Luxturna improves vision in the short term, as reported the New York Times Tuesday.

  • Jefferies analyst Michael Yee said the agency documents "look fairly benign and as expected," adding that he expects the advisory panel to back approval of the treatment.

  • FDA staff noted that due to the lack of data beyond one year, the long-term benefits of the therapy are unclear.

  • Spark is seeking approval of Luxturna for treating Leber congenital aumaurosis, which is caused by defects in the RPE65 gene.

  • Earlier this year, the FDA approved the drugmaker's request to alter the drug's proposed patient population to include any patient with an inherited retinal disease due to a biallelic RPE65 mutation.

  • If approved, Luxturna is expected to generate more than $400 million in annual revenue by 2021.

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