There continues to be a lot of activity and hope for patients suffering from , a rare and progressive fatal disease associated with restrictive cardiomyopathy and progressive heart failure. Although there are currently no approved therapies specifically indicated for treating TTR-CM, several pharmaceutical companies are in the late stages of drug development and getting closer to bringing therapies to market. Currently, health care professionals only manage the symptoms of the disease.
In the most recent news, Pfizer announced that their phase 3 ATTR-ACT study (which is evaluating tafamidis for the treatment of TTR-CM) met its primary endpoint, and demonstrated a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo. The preliminary safety data showed that tafamidis was generally well tolerated in this population and no new safety signals were identified.
There are two types of TTR-CM, a hereditary form and a wild-type form of the disease, which is not hereditary. The ATTR-ACT study included both types of patients. However, Alnylam and Ionis have developed their candidates, patisiran and inotersen, respectively, for hereditary TTR amyloidosis, or hATTR. Both patisiran and inotersen are under review in the EU and United States and could be launched this year.
Although the prevalence of transthyretin cardiomyopathy is unknown, it is estimated that less than 1% of people with the disease are diagnosed.
Tafamidis has been granted fast track status by FDA for TTR-CM. Meanwhile, tafamidis also has orphan drug designation for TTR-CM in both the EU and United States. Tafamidis is already approved for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) in 40 countries except United States.
