Skyline Therapeutics receives Orphan Drug Designation from FDA for SKG1108

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The US Food and Drug Administration has granted an Orphan Drug Designation (ODD) for SKG1108 from Skyline Therapeutics for the treatment of retinitis pigmentosa (RP).

SKG1108, a recombinant adeno-associated virus (rAAV) vector, is a one-time intravitreally delivered gene therapy utilizing the intravitreal capsid AAV.0106 to deliver single-stranded DNA encoding light-activatable proteins directly to the retina.¹ According to Skyline Therapeutics, the protein, which is regulated by “specific genetic elements,” aims to improve or restore visual function by generating new photo-sensing cells.

The company noted that ODD status for SKG1108 qualifies the drug for various benefits and will accelerate the development of the drug and “enhance patient access to the leading-edge therapeutic solution.”¹

ODD status encourages innovation and research in the development of treatments for rare diseases and makes these treatments available more quickly. The status qualifies drug sponsors for incentives including tax credits for qualified clinical trials, exemption from the prescription drug user fees, and potential 7 years of market exclusivity after approval.

RP, a type of inherited retinal disease (IRD), is linked to mutations in over 100 different genes or loci, all leading to progressive vision loss. The disease typically begins with the degeneration of rod cells, followed by the gradual loss of cone cells, and ultimately leads to the near-complete loss of all photoreceptor cells, leaving the retina non-functional and resulting in blindness. SKG1108, according to the company, compensates for the loss of rod and cone photoreceptors in patients with late-stage RP, agnostic on the specific gene or genetic mutation responsible for the condition.¹

References:

1. Skyline Therapeutics’ Novel Gene Therapy SKG1108 Receives FDA Orphan Drug Designation for Retinitis Pigmentosa. Press Release; September 2, 2024. Accessed September 2, 2024. https://www.skytx.com/news-2024_09_02