1. First gene therapy for rare blindness gets US FDA nod

First gene therapy for rare blindness gets US FDA nod

In a first, the US Food and Drug Administration has approved a novel gene therapy to treat children and adult patients with an inherited form of vision loss that may result in blindness.

By: | New Delhi | Published: December 20, 2017 3:07 PM
US FDA, FDA, us, US Food and Drug Administration, Food and Drug Administration Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision.

In a first, the US Food and Drug Administration (FDA) has approved a novel gene therapy to treat children and adult patients with an inherited form of vision loss that may result in blindness.

The new gene therapy titled “Luxturna (voretigene neparvovec-rzyl)” — a treatment for retinal dystrophy caused by a specific genetic mutation — is the first directly administered gene therapy approved in the US to treat a genetic disease other than cancer.

“The 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 Scott Gottlieb, Commissioner at FDA, in a statement late on Tuesday.

“I believe gene therapy will become a mainstay in treating, and, maybe, curing many of our most devastating and intractable illnesses,” he added.

“Biallelic RPE65” mutation-associated retinal dystrophy leads to vision loss and may cause complete blindness in certain patients.

The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision.

Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision.

Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore the patient’s vision loss.

“Patients with ‘biallelic RPE65’ mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed,” added Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research (CBER).

While, granting the approval of Luxturna to startup pharmaceutical company Spark Therapeutics Inc., the drug regulator stated that the gene therapy should be given only to patients who have viable retinal cells as determined by the physicians.

In 2017, FDA gave approvals for three gene therapy for patients with serious and rare diseases. In August, “Kymriah” was granted the approval to treats a form of leukemia and in October, “Yescarta” to treat a form of lymphoma.

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