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Human gene therapy products, like other medicines, may be eligible for an orphan drug status or designation provided sponsors can demonstrate that the product can treat a rare disease and that they can prove a scientific rationale basis for product’s effectiveness in treating the rare disease. If the eligibility criteria is met, then the orphan

Regulatory agencies across the globe are committed to ensuring that patients and the public have timely access to safe, effective, and high-quality medicines. They make significant developments, be it revision of guidelines to provide industry with robust support & guidance or expediting review and response times. Recently, the US FDA issued a guidance document for

Human gene therapy represents a groundbreaking frontier in medicinal products for treating complex diseases. This therapy aims to transform gene expressions or modify cellular properties for therapeutic purposes. Regulatory agencies support this innovation by ensuring the required guidelines are in place. For example, the US FDA stands as a guiding beacon for sponsors that wish

Advanced rare disease drug developers are companies or organizations that specialize in the research and development of treatments for rare diseases. Developing drugs for rare diseases poses unique challenges due to small patient populations, genetic variations, and the need for novel endpoint selection. In response, the FDA has allocated resources through initiatives like Prescription Drug