What Luxturna Could Mean for the Future of Gene Therapy

by | Nov 21, 2017

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In November, Sparks Therapeutics announced it had received unanimous approval from the FDA’s Cellular Tissue and Gene Therapy Advisory Committee for its new drug Luxturna (voretigene neparvovec). If the drug gains full FDA approval in January 2018, it will be the first ever gene cell therapy approved for an inherited disease. Even now, Luxturna has already generated a lot of talk in the pharmaceutical world.

Sparks describes Luxturna as a one-time-use gene therapy treatment designed to treat a rare pediatric eye disease. According to an article in Genetic Engineering and Biotechnology News, this disease is known as biallelic RPE65-mediated inherited retinal dystrophy. It is one of a group of inherited retinal diseases (IRD) — blinding conditions caused by one of more than 220 possible defective genes.

The Rare Disease Report site notes that, prior Luxturna, IRD was not considered to be treatable and that patients with this diagnosis suffered from symptoms such as night blindness and involuntary eye movements, and, eventually, total blindness. Therefore, one of the most important implications for the possible approval of this drug is the hope that it gives to children with this diagnosis.

In addition to the Phase III trial, which Sparks reports showed efficacy for 30 days after treatments as well as at four-year follow-up points, the company also submitted the results of two separate Phase I clinical trials, a natural history study, and a multi-luminance mobility test (MLMT), which was developed to assess visual function.

Sparks notes that, due to the nature of the drug and the disease being treated, Luxturna has received orphan drug, breakthrough therapy,and rare pediatric disease status and is now under priority review with the FDA. A final verdict is due in January 2018.

Possible Concerns

However, while the FDA generally follows the recommendations of the advisory committee, this is not a guarantee that FDA approval will be achieved. Genetic Engineering and Biotechnology News notes that some reviewers have already raised concerns. One of these concerns is how results like the MMLT tests relate to actual improved quality of life for patients. Another concern is the long-term evidence for efficacy: while Luxturna has been proven to improve certain markers — such as full field threshold light sensitivity and mobility test scores — overall visual acuity was not improved.

However, these concerns were not sufficient for the advisory committee, which noted in their commentary that there are no current pharmacological treatments for IRD and that the benefits of Luxturna outweigh the risks.

Luxturna and Gene Cell Therapy

As Sparks notes, Luxturna will make history if it does gain FDA approval, since it will be the first gene cell therapy to treat an inherited disease. However, earlier in the year, FDA approval was granted to two other gene cell therapies, Kymriah and Yescarta, both of which have been developed to treat specific forms of cancer. Many see these drugs as part of an important beginning in a new wave of disease treatments.

According to the Alliance for Cell and Gene Therapy, the MIT Technology review reports, gene cell therapy companies have raised nearly $1.2 billion in the second quarter of 2017, up 56 percent from last year. Currently, there are 504 gene cell trials underway across the globe, 34 of them in the final stages of study.

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