Pharmaceutical Insights: Delandistrogene moxeparvovec’s R&D Progress and its Mechanism of Action on Drug Target

Keywords:

Delandistrogene moxeparvovec, Delandistrogene moxeparvovec’s R&D Progress, Mechanism of Action for Delandistrogene moxeparvovec, drug target for Delandistrogene moxeparvovec.

Description:  

This article summarized the latest R&D progress of Delandistrogene moxeparvovec, the Mechanism of Action for Delandistrogene moxeparvovec, and the drug target R&D trends for Delandistrogene moxeparvovec.

 

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Delandistrogene moxeparvovec‘s R&D Progress

Delandistrogene moxeparvovec is an AAV-based gene therapy drug that targets micro-dystrophin. It falls under the therapeutic areas of Nervous System Diseases, Congenital Disorders, and Skin and Musculoskeletal Diseases. The drug is specifically indicated for the treatment of Muscular Dystrophy, Duchenne.

The originator organization of Delandistrogene moxeparvovec is Nationwide Children’s Hospital. It has reached the highest phase of development, which is Approved. The drug received its first approval globally on June 2023.

In terms of regulation, Delandistrogene moxeparvovec has undergone several processes. It has received Priority Review, indicating that the drug addresses an unmet medical need and has the potential to provide significant improvements over existing treatments. The drug also has a Paediatric investigation plan, which suggests that it has been studied in pediatric populations. Additionally, it has been granted Accelerated Approval, which allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. Delandistrogene moxeparvovec has also been designated as a Rare Pediatric Disease, indicating that it is intended to treat a rare disease affecting children. Furthermore, it has been granted Fast Track status, which expedites the development and review process of drugs. Lastly, the drug has been designated as an Orphan Drug, indicating that it is intended to treat a rare disease.

Please click on the image below to directly access the latest data (R&D Status | Core Patent | Clinical Trial | Approval status in Global countries) of this drug.

Mechanism of Action for Delandistrogene moxeparvovec: micro-dystrophin stimulants

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Drug Target R&D Trends for Delandistrogene moxeparvovec

Micro-dystrophin is a truncated version of the dystrophin protein found in the human body. It plays a crucial role in maintaining the structural integrity of muscle fibers. In individuals with Duchenne muscular dystrophy (DMD), a genetic disorder, the production of dystrophin is impaired, leading to muscle degeneration and weakness. Micro-dystrophin, when introduced into the body through gene therapy or other methods, aims to compensate for the lack of full-length dystrophin. It helps stabilize the muscle membrane, preventing muscle fiber damage and improving muscle function. The development and utilization of micro-dystrophin hold great promise in the treatment of DMD, potentially improving the quality of life for affected individuals.

According to Patsnap Synapse, as of 10 Oct 2023, there are a total of 2 micro-dystrophin drugs worldwide, from 9 organizations, covering 2 indications, and conducting 11 clinical trials.

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Conclusion

In summary, Delandistrogene moxeparvovec is an AAV-based gene therapy drug developed by Nationwide Children’s Hospital. It has been approved for the treatment of Muscular Dystrophy, Duchenne. The drug has undergone various regulatory processes, including Priority Review, Accelerated Approval, Rare Pediatric Disease designation, Fast Track status, and Orphan Drug designation. Its first approval globally occurred in June 2023.

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