The US Food and Drug Administration (FDA) has awarded Orphan Drug Designation to the experimental drug ALN-TTRsc02. Alnylam Pharmaceuticals Inc. is developing ALN-TTRsc02 as a possible treatment for transthyretin-mediated amyloidosis. The full article can be read here, at Business Wire.
Amyloidosis is a group of conditions that are caused by the build-up of amyloid protein throughout the body. This causes damage that can cause serious health problems, including, in some cases, organ failure. Transthyretin-mediated (ATTR) amyloidosis is a form of amyloidosis that occurs when amyloid deposits form as a result of abnormal versions of transthyretin, which is a blood protein usually involved in carrying vitamin A. The deposits can form in many areas of the body, including the heart, nerves, and digestive tract.
Two forms of ATTR amyloidosis are hereditary ATTR amyloidosis, and wild-type ATTR amyloidosis. The first hereditary, form of the condition is inherited from other family members who carry an altered TTR gene. It is estimated to affect 50,000 people worldwide. Generally, symptoms appear after the age of thirty. The wild-type form of the condition is not hereditary, and estimates of its prevalence are uncertain but thought to be around 200,000 people in the US and Europe. This form generally appears around or after the age of 65.
There is currently an unmet medical need for treatments of ATTR amyloidosis. The investigational drug ALN-TTRsc02 is a possible treatment for the condition. It functions by targeting and silencing certain messenger RNA molecules. This might stop the wild-type altered TTR protein from being produced, and reduce the formation of some amyloid deposits. However, more research is required to understand the effects of the experimental drug.
The Orphan Drug Designation by the FDA for ALN-TTRsc02 is hoped to support the further investigations of the drug. It has previously been designated an orphan medicinal product in the EU for treating transthyretin-mediated amyloidosis by the European Commission.