According to a story from Fierce Pharma, the drug company Alnylam Pharmaceuticals has seen its investigational therapy lumasiran (marketed as Oxlumo) recently gain approval for use in the EU as a treatment for primary hyperoxaluria type 1, a rare disease. This is a historic approval as outcomes in this illness are dire and there were no previously approved treatments. A decision on the medicine is expected in the US next month.
About Primary Hyperoxaluria
Primary hyperoxaluria is a rare progressive condition in which the body produces an excess of oxalate. This primarily leads to the development of deposits of oxalate that appear in the kidney and bladder, resulting in frequent kidney stones. There are three different types of primary hyperoxaluria which are distinguished by distinct causal mutations that affect different genes. The symptoms that result from these disorders are the result of oxalate deposits appearing in different areas of the body. Oxalate may appear in the bone marrow, joints, and bones. Frequent kidney stones result in symptoms such as acute pain, secondary infection of urine, and ultimately damage to the kidneys. As these stones appear early in life, kidney failure is of serious concern to primary hyperoxaluria patients. There aren’t any medications currently approved for this condition. Treatment approaches may include dialysis (as the kidneys fail) and kidney/liver transplant. To learn more about primary hyperoxaluria, click here.
The drug utilizes an RNAi mechanism that is capable of bringing down the excess production of oxalate. It is designed to be administered every three months subcutaneously. The key to maximizing the impacts of this new drug will be identifying patients more rapidly before the disorder has progressed and the kidneys shut down.
Access is Key
Rare disease therapies can often reach astronomical prices, and include some of the most expensive drugs on the planet. Alnylam aims to seek access arrangements that suit individual countries and account for local factors, with the overall goal being sustainable access to this therapy for patients, who are in dire need of a more effective intervention to prevent organ failure.