• 05 Sep 2024
• Admin
• News Article

FDA Grants ODD to Skyline Therapeutics’ Gene Therapy for Retinitis Pigmentosa

FDA Grants Orphan Drug Status to Skyline Therapeutics’ Gene Therapy for Retinitis Pigmentosa

Overview

Skyline Therapeutics made an announcement recently that the US FDA had accorded Orphan Drug Designation (ODD) to SKG1108, a novel gene therapy developed to treat Retinitis Pigmentosa (RP).

Orphan Drug Designation 

• Orphan drug designation (ODD) is a status granted to promising drugs or biological products by the FDA treating, preventing or diagnosing rare diseases. 
• The ODD program seeks to speed up access to these therapies by encouraging innovation and research for treatments against rare diseases. 
• ODD drugs qualify for benefits such as tax credits for clinical trials, exemption from prescription drug user fees, and potential seven years of market exclusivity upon approval.

SKG1108 for RP

This designation by FDA identifies SKG1108’s potential in curing RP and accords it a special status that includes several advantages that could facilitate its fast-tracking development and provision of this advanced therapy to patients.

rAAV: a Recombinant Vector

• Using rAAV, a recombinant vector that inserts into the host cell's genome, SKG1108 combines a viral vector within an eye that’s working outside its normal biological function in other tissues of the body. 
• It does so by sending DNA into the retina in such a way that it can produce proteins sensitive to light.

According to which genes these proteins are part of, they will help regenerate photo-sensing cells and substitute rod-cone photoreceptors that have been lost during advanced stages of retinal diseases. Notably, SKG1108 is made to be effective irrespective of the specific gene responsible for or involved in disease progression.

About Retinitis Pigmentosa

• Retinitis pigmentosa (RP) is a heritable disorder related to mutations in over one hundred different genes or loci leading to gradual loss of vision. 
• Although this varies from person to person, most individuals suffering from RP become legally blind by their forties. 
• Degeneration starts with death of rod photoreceptors followed by slow dying off cones ultimately resulting in almost complete blindness globally experienced today.

Current Treatments

Currently available treatments cannot prevent or reverse photoreceptor death across the board but may cater only to select patients based on particular gene changes while others remain out in cold when it comes to options available.

However, SKG1108's new methodology that seeks to enhance or restore visual function and acuity irrespective of any genetic defect holds great promise for broader populations of individuals with RP.