Gene Therapy for Hereditary Retinal Dystrophy Registered in Russia, Expanding Global Access

Gene Therapy for Hereditary Retinal Dystrophy Registered in Russia, Expanding Global Access

Good news for the inherited retinal disease (IRD) community! A therapy for hereditary retinal dystrophy, specifically Luxturna (voretigene neparvovec), has been officially registered in the Russian Federation. This development, reported by Новости GxP on April 28, 2022, marks an important step in making advanced treatments accessible to more patients worldwide who live with these challenging conditions.

This registration is particularly significant for individuals affected by biallelic RPE65 mutation-associated retinal dystrophy, a rare genetic condition that can lead to severe vision loss and even blindness. The availability of such a therapy in new regions offers hope for preserving and potentially improving vision for those who previously had limited treatment options.

Key Details of the Registration

According to Новости GxP, the EAEU (Eurasian Economic Union) registration was granted for Luxturna (voretigene neparvovec), a drug designed to treat hereditary retinal dystrophy. Luxturna's developer is Spark Therapeutics, a US-based company, with Novartis holding the license for distribution outside the United States.

Luxturna received approval from the U.S. Food and Drug Administration (FDA) in 2017 for the treatment of biallelic RPE65 mutation-associated retinal dystrophy. A few months later, the European Medicines Agency (EMA) also approved its use for the same indication. In Russia, hereditary retinal dystrophy was included in the list of diseases for which the state foundation, Circle of Kindness, procures therapies in the summer of 2021. The foundation's Board of Trustees approved the purchase of Luxturna as an unregistered drug in September 2021, and a contract for its supply was signed in November 2021 for six patients.

What This Means for Patients and Families

For patients and families living with RPE65-mediated retinal dystrophy, this registration signifies expanded access to a groundbreaking gene therapy. Luxturna is a one-time gene therapy that works by delivering a working copy of the RPE65 gene to retinal cells, aiming to restore functional vision and slow the progression of vision loss. This offers a new pathway for treatment in a region where such advanced therapies were not previously formally available. The Russian Ophthalmological Journal also reported on the first results of gene replacement therapy in Russia for children with biallelic RPE65 mutations, noting subjective improvements in visual perception, including better orientation in dark and twilight conditions, and improved contrast in all children.

Looking Ahead

The registration of Luxturna in the Russian Federation highlights the ongoing global effort to bring innovative gene therapies to individuals affected by IRDs. As more countries approve and integrate these treatments into their healthcare systems, the IRD community can look forward to a future with broader access to potentially life-changing interventions. This step underscores the importance of continued research, advocacy, and collaboration to ensure that no one with an inherited retinal disease is left without hope.