Ophthalmologists will be able to save the eyesight of American patients with retinal dystrophy with a special drug that will reach the first patients in the spring, and its price is impressive. The million dollar rumors for an ampoule turned out to be exaggerated, but only slightly.
The eyesight of American patients with retinal dystrophy caused by a confirmed bi-allelic RPE65 mutation can be saved by ophthalmologists using the recently approved FDA gene therapy – Luxturna (voretigene neparvovec-rzyl).
This is only the third gene therapy approved in the US. Treatment consists in a single injection – by injection – of the correct copy of the RPE65 gene into the cells of the retina. Injection requires special equipment and extreme precision.
Luxturna will go to the first patients in spring. The price has already been disclosed – it will be 425 thousand. dollars for a dose for one eye, or 850. for a double dose – administered to both eyeballs. Retinal dystrophy due to this mutation usually develops bilaterally, so most patients will require a double dose.
The price of the therapy is dizzying, but not record-breaking. In 2012, EMA approved the drug Glybera, intended for patients with lipoprotein lipase deficiency. Like Luxturna, it was a one-time gene therapy. Glybera initially cost $ 1,6 million, but in 2015 the price was reduced to $ 1 million. The drug was not selling very well, however. Only one patient received outside clinical trials. In spring 2017, the manufacturer withdrew from submitting the application for the extension of the expiring registration.
In a phase III clinical trial, Luxturna significantly improved vision in 27 of 29 participants. The manufacturer – Spark Therapeutics – estimates that 1000-1200 patients in the United States will benefit from the therapy.