Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) has received approval from the US FDA for ULTOMIRIS® (ravlizumab-cwvz) 100mg/mL formulation.
ULTOMIRIS® can reduce the time spent to receive treatment
The formulation has been approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It inhibits complement-mediated thrombotic microangiopathy in pediatric patients above one month and adult patients. ULTOMIRIS® 100mg/mL is an advancement in the treatment experience for aHUS and PNH patients because it minimizes average annual infusion by around 60% relative to ULTOMIRIS® 10mg/mL. At the same time, it delivers comparable efficacy and safety. Interestingly with ULTOMIRIS® 100mg/mL, PNH and aHUS patients will spend around 6 hours or less annually to receive treatment.
Jamile Shammo, a Medicine and Pathology Professor at Rush University Medical Centre, said that there have been significant clinical benefits of ULTOMIRIS® demonstrated in patients. This was shown through the completion of the C5 inhibition and also sustained efficacy. Shammo said that based on research evidence presented, the formulation has shown comparable efficacy and safety to ULTOMIRIS® 10 mg/mL with more benefit of considerably shorter infusion times.
PNH and aHUS have a poor prognosis
As a blood disorder, PNH is often characterized by complement-mediated red blood cell destruction, which can cause several deliberating symptoms and complications that include thrombosis. This can happen in the whole body and lead to organ damage and early death. On the other hand, aHUS can result in the progressive injury of vital organs, mostly the kidneys, through the rupturing of blood vessels’ walls and sometimes clots. In most cases, PNH and aHUS prognosis are poor, and as a result, accurate and timely diagnosis, including the right treatment, can be important in improving patient outcomes.
The company’s executive VP and R&D Head John Orloff said that ULTOMIRIS® is a leading treatment in the market for PNH, and it has also emerged as a standard of care therapy for aHUS. Orloff said that FDA approval is a massive step in developing a treatment for aHUS and PNH because it reduces infusion times.
