In collaboration with Scenic Biotech, Roche plans to conduct clinical trials for three rare disease treatments. The three drugs are treatments for Niemann-Pick disease type C (NP-C), Barth syndrome, and severe metabolic syndrome.
The FDA has rejected the first drug candidate for Alport Syndrome, bardoxolone, on the grounds of lack of efficacy. Despite this, researchers and the patient community consider this process to be a significant step towards the development of a treatment for Alport Syndrome.
The FDA has published a new draft guidance for gene-editing therapeutics. The guidance outlines what should be included in clinical trial applications for gene editing therapeutics and potential safety issues that should be tracked. In addition, long-term follow-up of at least 15 years after application of the treatment is recommended.
Chiesi Global Rare Diseases published a survey report showing that the economic burden of rare diseases is 10 times greater than that of common diseases (diabetes, heart disease, etc.). In addition, the annual economic burden for rare diseases for which there is no treatment was 21.2% higher than for rare diseases for which there is a treatment. Based on the results of the analysis, researchers say that government investment and expanded policy support are necessary to encourage the development of treatments for rare diseases.