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GenSight, a French biotechnology company, has announced the receipt of FDA orphan designation for retinitis pigmentosa gene therapy treatment in the US.

GenSight Biologics S.A. (Euronext: SIGHT) has announced the receipt of FDA Orphan Drug Designation in the US for its experimental retinitis pigmentosa (RP) treatment, “GS030”. The announcement follows a similar orhan designation grant in the EU, announced in Q4 of 2016. According to the company, GS030, is an optogenetic therapy delivered using adeno-associated viral (AAV) vectors to introduce the genetic sequence of a photosensitive protein into the nucleus of target cells. Once expressed, target cells should be able to function in a similar manner to photoreceptor cells enabling the restoration of vision in patients who are either blind due to RP, or have too few remaining photoreceptor cells to benefit from other potential therapeutic options. As the light sensitivity delivered to such cells is below the capacity of normal photoreceptors, the GS030 treatment additionally employs “biomimetic goggles” which, according to the company, “amplify the light signal at the appropriate wavelength to enable vision restoration”.


According to GenSight, orphan drug designation granted by the FDA, and by the European Commission, provides incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions designated as “rare”. The definition of an orphan disease changes depending on geography: a rare disorder in the US is one that affects less than 200,000 citizens, while in the European Uinion (EU), a rare disorder is one that affects up to five in 10,000 persons. There are between 5,000 and 7,000 orphan diseases listed in the literature and it is estimated that > 1 in 10 US citizens suffers from an orphan disease, while the comparable statistic for the EU is approximately 55 million people. Orphan drug designation is understood to provide commercial sponsors with financial incentives and benefits in the US and EU, including a period of market exclusivity if GS030 is approved for the treatment of retinitis pigmentosa patients. In a previous announcement, GenSight aslo stated that the EMA have granted “Advanced Therapy Medicinal Product” (ATMP) classification to GS030. The classification provides GenSight with scientific regulatory guidance from the EMA, notably on the applicable regulatory framework and pathway, as well as a centralized approval procedure in Europe.


Commenting on the achievement, Bernard Gilly, Chief Executive Officer of GenSight Biologics stated, “The Orphan Drug Designation both in Europe and in the United States, together with the Advanced Therapy Medicinal Product classification in Europe, fully recognize the urgent and unmet medical need for a safe and effective treatment for retinitis pigmentosa patients, and highlight the potential of optogenetics and GS030 to address it.”