Ruijian Medicine's NouvSight001 Receives FDA Orphan Drug Designation for Retinitis Pigmentosa

Mar 23, 2024 08:53 CST Updated 08:53

iRegene Therapeutics

Cell Therapy Product Developer

On March 20, 2024, NouvSight001, a breakthrough ophthalmic product developed by Wuhan iRegene Pharmaceutical Technology Co., Ltd. (iRegene), was granted Orphan Drug Designation (ODD) by the U.S. FDA. This product targets "a series of retinitis pigmentosa indications" and is the world’s first universal ophthalmic cell therapy product derived from induced pluripotent stem cells (iPSC), developed by the iRegene team based on their unique "AI + chemical induction" platform. It also represents a globally pioneering breakthrough in ophthalmic treatment. Retinitis Pigmentosa (RP) is a hereditary eye disease that can ultimately lead to blindness. Symptoms include night blindness and peripheral vision loss, which gradually worsens, potentially leading to tunnel vision (narrowed field of view) or even total blindness. RP can be caused by mutations in any of more than 50 related genes, with the pathogenesis primarily attributed to the progressive loss of rod cells and cone cells in the retina. Currently, there is no effective clinical treatment for RP.

Orphan Drug Designation (ODD) is a qualification granted by the FDA's Office of Orphan Products Development (OOPD) to drugs (including biologics) that meet the criteria for the prevention, treatment, and diagnosis of rare diseases. Drugs that obtain ODDAfter the drug is approved for marketing, it enjoys7-year marketing exclusivity, with tax deductions on expenses during the clinical research phase, and simultaneouslyExemptionNDA/BLA Application Fee (PDUFA).

This ophthalmic product NouvSight001F by the United StatesDAThe granting of orphan drug designation marks a significant milestone in iRegene's development. Regarding this, iRegene...Dr. Cai Meng of MO stated that the FDA's orphan drug designation for the company's ophthalmic product NouvSight001 demonstrates the strategic foresight in the company’s early pipeline planning and indication selection strategy. It also recognizes the uniqueness of this product in the field of ophthalmic retinal-related disease treatment and its superior therapeutic mechanism. This orphan drug designation will play a significant role in accelerating the subsequent commercialization process of the product. iRegene will also gain market exclusivity upon the product's market launch, enhancing the commercial value of the pipeline. The company will subsequently strive to advance the registration and clinical trials of the product to benefit patients worldwide as soon as possible. Emmanuel Montet, SVP of International Affairs at iRegene, stated that the granting of this Orphan Drug Designation (ODD) proves that iRegene's innovative spirit and relentless efforts have once again achieved an important milestone. This achievement is also a result of the team’s rapid learning ability, hard work, and dedication, bringing iRegene to this critical moment. The team's drive for excellence further strengthens iRegene's determination to address the challenges faced by patients with retinitis pigmentosa.

iRegene, as one of the earliest biotech companies globally to apply "AI + chemical induction" to the specific functional modification of cells, has established a rich pipeline of universal iPSC products on this unique platform. It aims to develop universal cell therapy products and provide new treatment options for currently "incurable" diseases such as neurodegenerative disorders represented by Parkinson's disease and blindness. Relying on its self-developed platform based on "AI + chemical induction" for the precise directional modification of cell functions, iRegene determines induction protocols for derivative cells through accurate differential analysis, enabling the efficient transformation of induced pluripotent stem cells (iPSCs) and ensuring drug feasibility. The advantages of the "AI + chemical induction" platform have been validated in clinical stages. Its first universal cell therapy product, NouvNeu001, the world’s first universal chemically induced product for Parkinson’s disease, was approved to enter combined Phase I-II clinical trials in August 2023. The first patient was dosed in January 2024, and so far, promising efficacy and safety data have been obtained in registered clinical trials.

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Disclaimer: The content is sourced from iRegene. This article aims to share knowledge, and all content is for reference only, not constituting any advice.