Spark Therapeutics, a drug developer spun out of Children’s Hospital of Philadelphia, has been granted breakthrough therapy designation. The US Food and Drug Administration (FDA) granted the designation to the spin-out’s lead drug candidate to prevent blindness.
Called Spk-Rpe65, the drug candidate is still experimental. It is hoped that it could treat inherited retinal dystrophies, a range of conditions affecting the eye ultimately leading to blindness.
The FDA designation signifies expedited development and review for Spark’s drug candidate. It is given to experimental drugs when said therapy is deemed to be significantly improving patient’s odds over existing therapies. There is currently no treatment available for the range of conditions targeted by Spk-Rpe65.
The announcement is good news for Spark’s investors who put $72.8m into the company in May 2014.
Children’s Hospital of Philadelphia is affiliated with Pennsylvania University’s School of Medicine. The spin-out is currently moving its headquarters to University City Science Centre.
Jeffrey Marrazzo, co-founder and chief executive at Spark, said: “FDA’s breakthrough therapy designation for Spk-Rpe65 underscores the serious unmet medical needs faced by patients with genetic blinding conditions.”
