[Monoclonal antibodies for monogenic diseases: a 2019 update]

Monogenic diseases are rare genetic diseases but they are numerous and display a highly variable degree of severity. First uses of monoclonal antibodies to treat monogenic diseases started in the 2000's and many clinical trials are ongoing. Anti-IL-1β therapies have greatly modified the outcome of auto-inflammatory diseases by modulating inflammatory response and reducing the risk of secondary amyloidosis. Anti-TNF-α are also used in such diseases. In atypical hemolytic and uremic syndrome due to deficiencies in the control of alternative complement pathway, eculizumab, an anti-C5 monoclonal antibody, has improved renal outcome in treated patients. More recently, lanadelumab, an anti-plasma kallikrein antibody, has reinforced the therapeutic arsenal in hereditary angioedema and burosumab, anti-FGF23, that of X-linked hypophosphatemia. Such examples reflect the importance of monoclonal antibody therapy of monogenic diseases, the interest of considering such an option as well as the need for future researches.