Haemophilia, state of the art and new therapeutic opportunities.
Abstract
Haemophilia A and B are rare bleeding disorders. Over the past decades,
they have been transformed from debilitating diseases to manageable
conditions. However, several challenges and unmet needs remain in the
treatment of the haemophilia limiting the QoL of the patients. These
challenges are now being addressed by EHL recombinant factors,
rebalancing and substitution therapies. Gene therapy and genome editing
show promise for a definite clinical cure. Herein, we provide an
overview of new therapeutic opportunities for haemophilia and their
advances and limitations. The database on human medicines from EMA was
used and data from rare disease (orphan) designations and EPARs were
retrieved for the analysis. Clinical Trial databases were used to query
all active studies on haemophilia. Gene therapy medicinal products based
on AAV and lentiviral vectors are in development and clinical trials
have reported substantial success in ameliorating bleeding tendency in
haemophilia patients. The prospect of gene editing for correction of the
underlying mutation is on the horizon with considerable potential. We
are entering an era of innovation and abundance in treatment options for
those affected by bleeding disorders but issues still remain about the
affordability and accessibility to patients, the long-term durability,
safety and efficacy.