Maribavir is being developed for the treatment of cytomegalovirus (CMV) infections and the first indication expected for maribavir is for patients that are clinically refractory and/or genetically resistant to ganciclovir (GCV), valganciclovir (VGCV), cidofovir (CDV) and foscarnet (FOS). CMV is a common virus that usually only causes mild infection. In people with weakened immunity, such as transplant patients receiving treatment that reduce the activity of the immune system, CMV can cause serious infection such as pneumonia, colitis, hepatitis and retinitis. Refractory or resistant CMV in patients with impaired immunity is a long-term debilitating and life-threatening condition and associated with higher death rate in transplant patients.
Maribavir, administered orally, is thought to block the action of an enzyme of the virus called UL97 kinase. By blocking the enzyme, the medicine is expected to prevent viruses from reaching maturity, so that no new infectious viruses can be produced. If licensed, maribavir would offer an alternative treatment option for patients with CMV infections that are clinically refractory and/or genetically resistant to GCV, VGCV, CDV or FOS after stem cell or solid organ transplantation.
Deferiprone is in clinical development for patients with sickle-cell disorder (SCD) and other anaemias that are suffering from iron overload due to frequent transfusions to increase their red blood cell count. SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle red blood cells die early, and patients often require blood transfusions. Iron overload is an effect of frequent transfusions in SCD. Excess iron in the body can be toxic to major organs like the heart and liver.