Luxturna Approved to Treat Retinal Dystrophy
December 19, 2017 – The U.S. FDA has approved Luxturna™ (voretigene neparvovec-rzyl), manufactured by Spark Therapeutics, to treat individuals with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Luxturna is the first FDA-approved gene therapy to treat a hereditary disease caused by mutations in a specific gene. It is the third gene therapy to win FDA approval in the U.S.
A hereditary retinal disorder, biallelic RPE65 mutation-associated retinal dystrophy affects an estimated 1,000 to 2,000 Americans. The disease results from a mutation in the RPE65 gene that reduces or eliminates the production of an enzyme necessary for vision. The affected individual’s vision becomes progressively worse over time, usually beginning in childhood or adolescence, and leading to complete blindness. Luxturna uses a modified, naturally occurring virus to deliver a normal copy of the RPE65 gene to retinal cells. This allows the cells to produce the needed enzyme to improve the patient’s vision. In clinical studies, patients treated with Luxturna showed significant improvement one year post-treatment.
Luxturna must be administered by a surgeon experienced in intraocular surgery, and is a one-time treatment. Recommended dosing is a single 0.3mL subretinal injection delivered to each eye on separate days, no less than six days apart. A short course of systemic oral corticosteroids, lasting a total of seventeen days, should be used to help prevent a possible immune response to Luxturna.
The actual wholesale price (AWP) for Luxturna is $510,000 per eye, for a total of $1.2 million if both eyes require treatment. Launch is expected during the first quarter of 2018.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.