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Genetic and Congenital
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Gene therapy remains challenging for rare diseases

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Advanced therapy medicinal products (ATMPs) and gene therapies have been attracting attention in the field of rare diseases. However, shortcomings in safety and efficacy data make the adoption of these therapies questionable.

A recent review found that as of 2019 there are 8 gene therapies approved in the United States and/or European Union. These approvals were based on completed and ongoing pivotal clinical trials, however, the small sample sizes used in the pivotal clinical trials raise concerns.

Questions related to efficacy, incomplete trials, and serious adverse events are challenges the study authors indicated may result in some of these treatments being used a last-line therapy.

Reference
Balkhi B. Efficacy, safety and cost of gene therapy medicinal products in the U.S. and Europe. Drugs Today (Barc). 2022 May;58(5):223-240. doi: 10.1358/dot.2022.58.5.3331005. PMID: 35535814.

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