From Wikipedia, the free encyclopedia
Gene therapy medication
Voretigene neparvovec , sold under the brand name Luxturna , is a
gene therapy medication for the treatment of
Leber congenital amaurosis .
[4]
Leber's congenital amaurosis, or biallelic
RPE65 -mediated inherited
retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition.
[7] The gene therapy is not a cure for the condition, but substantially improves vision in those treated.
[8] It is given as a subretinal injection.
Voretigene neparvovec was approved for medical use in the
United States in December 2017,
[9]
Australia in August 2020
[10] and in
Canada , in October 2020.
[11] It is the first in vivo gene therapy approved by the US
Food and Drug Administration (FDA).
[12]
Medical uses
Voretigene neparvovec is
indicated for the treatment of people with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
[6]
Chemistry and production
Voretigene neparvovec is an
AAV2
vector containing human
RPE65
cDNA with a modified
Kozak sequence . The virus is grown in
HEK 293 cells and purified for administration.
[13]
History
It was developed by
Spark Therapeutics and
Children's Hospital of Philadelphia .
[14]
[15]
[16]
It was granted
orphan drug designation for Leber congenital amaurosis and
retinitis pigmentosa .
[17]
[18] A
biologics license application was submitted to the US
Food and Drug Administration (FDA) in July 2017 with
Priority Review .
[7]
Phase III clinical trial results were published in August 2017.
[19] On 12 October 2017, a key advisory panel to the FDA, composed of 16 experts, unanimously recommended approval of the treatment.
[20] The FDA approved the drug in December 2017.
[9]
[5] With the approval, Spark Therapeutics received a
pediatric disease priority review voucher .
[21]
The first commercial sale of voretigene neparvovec, which was also the first sale of any gene therapy product in the United States, occurred in March 2018.
[22]
[12] The price of the treatment at the time was announced as being $425,000 per eye.
[23]
References
^
a
b
"Luxturna Australian Prescription Medicine Decision Summary" . Therapeutic Goods Administration (TGA) . 13 August 2020. Retrieved 16 August 2020 .
^
"Luxturna Product information" . Health Canada . 25 April 2012. Retrieved 21 October 2020 .
^
"Summary Basis of Decision (SBD) for Luxturna" . Health Canada . 23 October 2014. Retrieved 29 May 2022 .
^
a
b
"Luxturna- voretigene neparvovec-rzyl kit" . DailyMed . 4 December 2019. Retrieved 14 August 2020 .
^
a
b
"Luxturna" . U.S.
Food and Drug Administration (FDA) . 19 December 2017. Retrieved 2 April 2020 .
^
a
b
"Luxturna EPAR" .
European Medicines Agency (EMA) . 24 September 2018. Retrieved 21 October 2020 . Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^
a
b
"Press Release - Investors & Media - Spark Therapeutics" . Ir.sparktx.com . Retrieved 9 October 2017 . [
permanent dead link ]
^ McGinley L (19 December 2017).
"FDA approves first gene therapy for an inherited disease" . Washington Post .
^
a
b
"FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss" . U.S.
Food and Drug Administration (FDA) . 24 March 2020. Retrieved 28 November 2022 .
^
"Luxturna" .
Therapeutic Goods Administration (TGA) . 13 August 2020. Retrieved 22 September 2020 .
^
" 'I never saw stars before': Gene therapy brings back 8-year-old Canadian boy's sight" . CTVNews . 14 October 2020. Retrieved 21 October 2020 .
^
a
b
"First Gene Therapy For Inherited Disease Gets FDA Approval" . NPR . 19 December 2017.
^ Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. (August 2017).
"Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial" . Lancet . 390 (10097): 849–860.
doi :
10.1016/S0140-6736(17)31868-8 .
PMC
5726391 .
PMID
28712537 .
^
"Spark's gene therapy for blindness is racing to a historic date with the FDA" . Statnews.com . 9 October 2017. Retrieved 9 October 2017 .
^ Clarke T.
"Gene Therapy for Blindness Appears Initially Effective, Says U.S. FDA" . Scientific American . Retrieved 12 October 2017 .
^
"FDA approves Spark's gene therapy for rare blindness pioneered at CHOP" . Philly . Retrieved 24 March 2018 .
^
"Voretigene neparvovec - Spark Therapeutics - AdisInsight" . adisinsight.springer.com .
^ Lewis R (13 October 2017).
"FDA Panel Backs Gene Therapy for Inherited Blindness" . Medscape .
^ Lee H, Lotery A (August 2017). "Gene therapy for RPE65-mediated inherited retinal dystrophy completes phase 3". Lancet . 390 (10097): 823–824.
doi :
10.1016/S0140-6736(17)31622-7 .
PMID
28712536 .
S2CID
26983863 .
^
"Landmark Therapy to Treat Blindness Gets One Step Closer to FDA Approval" . Bloomberg.com . 12 October 2017. Retrieved 12 October 2017 .
^
"Spark grabs FDA nod for Luxturna, a breakthrough gene therapy likely bearing a pioneering price" . FiercePharma . 19 December 2017.
^
"The anxious launch of Luxturna, a gene therapy with a record sticker price" . STAT . 21 March 2018. Retrieved 24 March 2018 .
^ Tirrell M (3 January 2018).
"A US drugmaker offers to cure rare blindness for $850,000" . CNBC. Retrieved 3 January 2018 .
Further reading
External links