Spark Therapeutics’ injectable therapy waits for FDA approval

Date: 2017-10-16   Author: Saipriya Iyer  Category: #industry

Spark Therapeutics’ injectable therapy waits for FDA approval

A panel of federal health advisers has recently endorsed an experimental approach for patients affected with hereditary blindness, setting the stage for the FDA approval of a groundbreaking gene therapy treatment. Reportedly, a board of experts from the Food and Drug Administration have unanimously voted in favor of the ‘Spark Therapeutics' therapy, which aims to improve the vision in patients affected with a rare mutation that ultimately destroys normal eyesight. The vote expanse demands a recommendation for the therapy approval, cite experts. It is not usually a mandate for the FDA to follow the panel’s recommendation, though the organization usually concedes to it such endorsements. As per reliable sources, the FDA has a couple of months in hand until the middle of January 2018 to give the final verdict.

Manufactured by the Philadelphia-based pharmaceutical giant, Spark Therapeutics, the treatment involves injecting a healthy edition of RPE65 gene to patients who hereditarily possess defective pairs of the gene. Reportedly, this injectable therapy would allow the patients to release a specific protein in the body that is primarily responsible for eyesight. Though the treatment does not assure a perfect vision, it does depict substantial improvements, claim experts. Also, it has been reported that if approved, it would be the first gene therapy in U.S. for inherited disease, offering hope to patients with a variety of related conditions.

While the FDA has given approval to Kymriah, the gene therapy treatment meant for childhood leukemia, prior to this, Spark’s Luxturna is claimed to be the first true gene therapy that the FDA had approved. As per reliable sources, around 1000 to 2000 Americans suffer from retinal disorder caused by mutated RPE65 gene. While it is still to be watched out whether this Spark Therapeutics’ injectable therapy receives the much-awaited FDA approval, experts believe, it would certainly pave way for other generic treatments with regards to a variety of inherited disorders.



About Author


Saipriya Iyer linkdin-boxtwitter

Saipriya Iyer

Saipriya Iyer currently works as a content developer for AlgosOnline. A computer engineer by profession, she ventured into the field of writing for the love of playing with words. Having had a previous experience of 3 years under her belt, she has dabbled with website...

Read More

Post Recommendents

Innovations in surveillance system—a growth enabler in air traffic management

Author: Saipriya Iyer

Transforming air traffic management into a more interoperable, automated, modular and scalable system will potentially reap rewards from developments in virtualization and digital technologies. Seamless high-performing...


Stents industry to witness robust technological innovations with ongoing R&D activities

Author: Pankaj Singh

One of the fastest emerging verticals within the healthcare and medical devices space, the global stents market is set to witness numerous interesting trends in the coming years. Ongoing research and development in the...


Amazon India launches ‘Select’ program to assist emerging brands

Author: Shikha Sinha

E-commerce giant Amazon has reportedly started a new program called ‘Select’ in India. As per trusted sources, the program will enable emerging brands in the country to get access to a suite of brand buildi...