Regenxbio's RGX-202 Gene Therapy for Duchenne Muscular Dystrophy Receives Fast Track Designation from FDA

Regenxbio Inc. has received a fast track designation from the FDA for its innovative gene therapy, RGX-202, aimed at treating Duchenne muscular dystrophy. This therapy has been designed to deliver and express genes in the heart and skeletal muscle by utilizing the NAV AAV8 vector and a muscle-specific promoter. The FDA had earlier granted the orphan drug designation and rare pediatric disease designation for this therapy.

Publish Date: 11-04-2023 Source: Regenxbio Inc.

Duchenne muscular dystrophy (DMD) is caused by a lack of dystrophin, a protein that aids in the maintenance of muscle cells. Symptoms usually appear in early childhood, between the ages of 3 and 5. The disease primarily affects boys but can also affect girls in rare cases. Because the gene that can carry a DMD-causing mutation is on the X chromosome, DMD is inherited in an X-linked pattern. Mutations in the DMD gene cause progressive muscle weakness, loss of independent ambulation by early adolescence, and premature death from cardiorespiratory complications.

The worldwide prevalence of DMD was 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population. Additionally, the global DMD birth prevalence was 19.8 per 100,000 live male births.

However, the current Duchenne Muscular Dystrophy (DMD) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (SRP-4045, TAS-205, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Duchenne Muscular Dystrophy treatment. The key companies in the advanced development stage are Sarepta Therapeutics, Inc., Taiho Pharmaceutical Co., Ltd., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Duchenne Muscular Dystrophy (DMD) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2021 to 2032.

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