First gene therapy could hit the market in January
- Spark Therapeutics announced Monday morning that its application for approval for its gene therapy for blindness has been accepted by the Food and Drug Administration.
- The company was given a user fee goal date of Jan. 12, 2018 for voretigene neparvovec, a treatment for patients with vision loss due to confirmed biallelic RPE65-mediated inherited retinal disease (IRD).
- Spark has proposed the trade name of Luxturna. The FDA evaluates trade names to make sure they do not look or sound like other drug names in order to avoid potential mix-ups.
The first gene therapy could hit the market as early as January. Spark submitted its Biologics Licensing Application earlier this year and announced the regulatory agency has accepted the application, as well as granted accelerated approval.
Luxturna is a potential one-time treatment — verging on a cure — for a rare genetic form of blindness caused by a gene mutation.
There have been a small handful of gene therapies approved in Europe, but they have not performed well commercially. The industry is still trying to find a pricing model that could work for a one-time, potentially curative treatment.
Spark will be watched closely to see how it handles the pricing of Luxturna.
The company is a pure-play gene therapy company that has several other gene therapies in development, including a highly anticipated medicine for the treatment of hemophilia that is in partnership with Pfizer.
Luxturna was tested in 41 patients across a range of age groups and showed statistically significant improvement in vision compared with the control group. Patients continued to show functional gains in vision two years after the administration of the drug.
- BioPharma Dive Alternative pricing models a fantasy for gene therapies?
- Spark Therapeutics Statement
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