US FDA to decide on Spark's vision loss gene therapy Luxturna in January
Spark announced it had been given a PDUFA date of January 12, 2018 in a US Securities Exchange Commission (SEC) filing this week.
Luxturna (voretigene neparvovec) is a gene therapy intended to treat patients with vision loss caused by an inherited retinal disease.
Data from a Phase III trial of the drug recently published in the Lancet suggest it "improved functional vision in RPE65-mediated inherited retinal dystrophy previously medically untreatable."
The US FDA’s decision to assign Luxturna priority review status – which cuts four months of the standard review timeline – could see it become the first gene therapy to be approved in the country.
Manufacture
A Spark told us Luxturna is made at a facility the firm has built in West Philadelphia.
A spokeswoman explained that: "Our manufacturing platform was developed by scientists who have a track record of identifying appropriate disease targets as well as overcoming obstacles to safe and efficient gene transfer into specific target tissues.
"This industry-leading platform can produce adeno-associated viral (AAV) and lentiviral-based vectors. For our first investigational gene therapy, Luxturna, we manufacture small batches for an ultra rare disease, using roller bottles."
Gene genie or hard cell?
The US FDA defines a gene therapy as a product which introduces “genetic material into a person’s DNA to replace faulty or missing genetic material, thus treating a disease or abnormal medical condition.”
Under this definition Luxturna is definitely a gene therapy.
In contrast, Novartis AG’s blood cancer treatment tisagenlecleucel, which an FDA review panel recommended for approval last week, is not.
Tisagenlecleucel is a cell therapy. Nevertheless, Reuters, the Associated Press and other publications reported that the Novartis product could be the first gene therapy approved in the US.
The Spark spokeswoman emphasised the difference, telling us "Luxturna corrects an underlying genetic defect. One goal of gene therapy research is to determine whether a new or functional gene can be used to restore the function of, or inactivate, a mutated gene.
"CAR-T cell therapy is a therapy for an acute condition, not a genetic disease. Immunotherapy utilizes gene transfer to reprogram the patient's T cells to target something new, leukemia antigens in the case of Novartis."
