According to a story from BioSpace, the biopharmaceutical company Dicerna Therapeutics, Inc. has announced recently that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to its experimental drug nedosiran. This investigational product candidate is being developed as a treatment for primary hyperoxaluria, a rare disease. Dicerna focuses on the development of RNA interference therapies.
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.
About Rare Pediatric Disease Designation
Rare Pediatric Disease designation is a special program from the FDA. It is reserved for drugs developed for rare diseases that primarily affect people 18 years or younger. A rare disease is defined as any disease that affects less than 200,000 people in the US. The disease must be life-threatening and serious. The designation confers a Priority Review voucher to the recipient company. This voucher can be used on a later drug application or can also be sold to another pharmaceutical company.
The presentation of this designation to nedosiran is a reflection of the dire state of treatment for primary hyperoxaluria, which has no approved treatments; patients often live with the expectation that they will eventually face kidney failure and kidney disease, ultimately requiring dialysis or even a kidney transplant. There is an urgent need for more effective, disease-modifying treatments.
Nedosiran is classified as an inhibitor of lactate dehydrogenase enzyme LDH, utilizing Dicerna’s proprietary RNA interference technology platform. It is currently being evaluated for all three types of primary hyperoxaluria.