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Alnylam has recently completed the third phase of their clinical trial of lumasiran, which was created for the treatment of primary hyperoxaluria type 1 (PH1). While they will not release detailed results until March of 2020, the company stated that this drug did meet all of the primary and secondary goals. As there are no FDA approved treatments for this disease, researchers are very excited about the possibility of this drug being the first. They hope that their RNAi drugs will not only better the lives of those with PH1, but that they will be able to apply these advancements to other diseases.
About Primary Hyperoxaluria Type 1 (PH1)
Primary hyperoxaluria type 1 (PH1) is a rare disease that affects the kidneys. It is characterized by the buildup of oxalate, which normally leaves the body with urine after being filtered by the kidneys. In this disease oxalate accumulates in the kidneys and urinary tract. It then combines with calcium to create calcium oxalate, which is the main component of kidney stones.
A mutation in the AGXT gene causes this disease. This gene creates the enzyme alanine-glyoxylate aminotransferase, and the mutation prevents the breakdown of glyoxylate. If this substance accumulates, it leads to oxalate, which damages the kidneys and other organs.
The main symptom of PH1 is kidney and bladder stones. Other symptoms include stunted growth, kidney failure, and renal failure.
In order to diagnose this disease a clinical examination, identification of symptoms, and study of patient history are necessary. After these are all completed a diagnosis may be confirmed through blood tests, imaging tests, molecular genetic testing, or a biopsy.
Treatment of PH1 focuses on lowering the levels of calcium oxalate and ensuring that renal function remains intact. Because the kidneys degenerate as time passes, early diagnosis and treatment are important. Treatments include oral potassium citrate, drugs like thiazides or pyridoxine, avoiding large amounts of vitamins C and D, dietary calcium supplements, and drinking large amounts of fluids. If damage does occur to the kidneys, dialysis and a transplant may be necessary.
About Alnylam’s Study
Alnylam recently completed the third phase of their study of lumasiran, which is an RNAi drug. The study included about 30 participants and based its success off of the levels of oxalate excreted in urine. This success has led them to seek approval of lumasiran from the FDA. Alnylam will also conduct two more Phase 3 studies in order to evaluate this drug’s reach. One of these studies will examine the effects on people younger than six, while the other will look at people of all ages who have PH1 and advanced renal disease.
Other companies, such as Dicerna, Allena Pharmaceuticals, and OxThera, are also developing treatments for this disease. As people with PH1 have had very limited options in treatment for a long time, this work in treatment for this disease is very exciting.
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