Un interesante autorizado por la FDA: LUXTURNA

Aquí el BLA APROVAL de voretigene neparvovec-rzyl (LUXTURNA) de Spark Therapeutics, Inc. de fecha 19 de diciembre de 2017.

Aquí el Package insert  del que extraigo:

1 INDICATIONS AND USAGE

 LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.

 Patients must have viable retinal cells as determined by the treating physician(s). 

Aquí el Summary Basis for Regulatory Action

 LUXTURNA is a recombinant adeno-associated virus serotype 2 (AAV2) vector expressing the gene for human retinal pigment epithelium 65 kDa 5 protein (hRPE65), for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
The RPE65 protein is expressed in retinal pigment epithelial (RPE) cells and converts all-transretinyl ester to 11-cis-retinol, which subsequently forms the chromophore, 11-cis-retinal, in the retinoid visual cycle. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 isomerohydrolase activity, blocking the visual cycle and resulting in impairment of vision. LUXTURNA is designed to deliver a normal copy of the gene encoding the human RPE65 to cells of the retina in persons lacking normal function of RPE65.

This document summarizes the basis for regular approval for LUXTURNA. A Phase 1 clinical study and a Phase 3 clinical study provide the primary evidence of safety and effectiveness for the BLA submission. The recommendation for approval is based on the improvement in the multi-luminance mobility testing (MLMT) demonstrated in the Phase 3 study. The more serious risks of LUXTURNA include endophthalmitis (infection inside of the eye), permanent decline in visual acuity, increased intraocular pressure, retinal abnormalities (e.g. retinal tears or breaks), and cataract development and/or progression.

12 años van desde el primer trámite a la autorización

Sobre la fabricación se restringe el acceso a información que la empresa considera no debe publicitar (página 7)

Respecto del precio y de la financiación  Fiercepharma dice:

There's a new medicine atop of pharma's global pricing charts, and it's Spark Therapeutics' Luxturna. After winning FDA approval in December, the company said Wednesday its gene therapy will cost $850,000, or $425,000 per eye before discounts. 

 To soften the blow, Spark is introducing outcomes-based deals with Harvard Pilgrim and Express Scripts affiliates—and it's in talks for more. Under those arrangements, the company won't collect full payment if its superpricey drug doesn't work. The company is also working on a proposal with the Centers for Medicare and Medicaid Services that would allow payments over multiple years.