'Luxturna' recommendation good for all AAV based gene therapies says advocacy group

By Gareth Macdonald contact

- Last updated on GMT

Gettyimages/cosmin4000
Gettyimages/cosmin4000

Related tags: U.s. securities and exchange commission

The US approval recommendation for Spark Therapeutics’ blindness gene therapy voretigene neparvovec is also an endorsement of AAV delivery technologies according to the Alliance for Regenerative Medicine.

A US Food and Drug Administration (FDA) advisory committee recommended that voretigene neparvovec be approved to treat biallelic RPE65-mediated inherited retinal dystrophy (IRD) last week, basing the decision on Phase III data showing it improved vision in 93% of recipients.

The therapy – which has been given the provisional name Luxturna - is a recombinant adeno-associated viral (AAV) vector serotype 2, expressing the gene for human retinal pigment epithelial 65 kilodalton protein (AAV2-hRPE65v2).

The Food and Drug Administration (FDA) is due to make a final decision in January.

Patient groups and financial analysts​ welcomed the recommendation.

AAV endorsement

The Alliance for Regenerative Medicine (ARM), a US advocacy group that lobbies on behalf of advanced therapy manufacturers, was also positive about the endorsement.

A spokeswoman told us "ARM believes this endorsement serves as an acknowledgement of the profound potential of gene therapy​."

"This, combined with the FDA’s recent approval of Novartis’s cell-based immunotherapy CAR-T product, Kymriah, and the agency’s implementation of the new RMAT designation for cell and gene therapies, is additional evidence of the growing awareness and validation of this sector and the significant patient benefits​." 

According to ARM, at present 504 gene therapies are in clinical development around the world, 34 of which are in Phase III clinical trials.

Price?

Spark has not said how much it will charge for Luxturna. According to analysts, the single-shot treatment may cost as much as $1m​.

According to a document​ Spark filed with the Securities Exchange Commission (SEC) in February, “there are approximately 3,500 individuals with RPE65 -mediated IRD in the United States, as well as France, Germany, Italy, Spain and the United Kingdom​.”

Related news

Show more

Related products

show more

Single-Step Influenza and Dengue Virus Purification

Single-Step Influenza and Dengue Virus Purification

Bio-Rad Laboratories | 08-Oct-2018 | Application Note

CHT XT is the newest addition to the CHT family of media. It has been designed for superior physical robustness to ensure it can be used repeatedly over...

Cultivation of 3D Cell Aggregates in Perfusion

Cultivation of 3D Cell Aggregates in Perfusion

Eppendorf for Bioprocess – Solutions that grow with you | 17-Sep-2018 | Application Note

3D cell aggregates are of great interest for applications like disease modeling and manufacturing of stem cell-based products. Read more about the cultivation...

Downstream Purification of Large Biomolecules

Downstream Purification of Large Biomolecules

Bio-Rad Laboratories | 17-Sep-2018 | Research Study

Our newest strong anion exchange resin provides both high binding capacity and
high recovery, even at high flow rates. A rigid bead complete with...

Nifty Biosimilar

Nifty Biosimilar

UGA Biopharma | 13-Mar-2018 | Research Study

Aflibercept (Zaltrap®) is a recombinant fusion protein with complex post-translational modifications. Therefore deep understanding of biosimilar development...

Related suppliers

Follow us

Products

View more

Webinars