Treatment of PBC-A step forward.

Recent years have seen a growing interest in PBC within the scientific community, healthcare providers and industries, coupled with great advances in the understanding of the molecular and genetic basis and the natural history of the disease. Several disease-modifying agents targeting the immune-mediated response and bile-acid therapies are at different stages of development, some with promising results. A new drug, obeticholic acid, has been recently registered in the US and Europe as a second-line treatment in refractory PBC. International cohort studies have highlighted a disease heterogeneity, and so the need to provide patients with a more personalised management based on their risk of disease progression. Major challenges remain the development of surrogate endpoints in clinical trials acceptable to regulatory authorities, in a disease with a relatively low rate of events; and the development of clinical tools for patient's risk stratification and selection of those with greatest potential benefit from second-line therapies.