dm+d

Unassigned

New Medicines

Information

New molecular entity
AstraZeneca
Ionis Pharmaceuticals

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

Ligand conjugated antisense (LICA) drug which inhibits the production of transthyretin. Antisense is a non0coding DNA strand of a gene - it is a way of silencing genes.
Amyloidosis is a hereditary, systemic disease due to TTR gene mutations. Mutations cause abnormal amyloid proteins to accumulate & damage body organs /tissues, causing intractable peripheral sensory and autonomic neuropathy, and/or cardiomyopathy. Familial amyloidosis caused by a transthyretin mutation occurs in ~ 1 in 100,000 Caucasians in the USA and ~4% in African Americans. Symptoms usually begin between 40 and 65 years of age[4]. ~10,000 people worldwide have polyneuropathy due to hATTR [5].
Transthyretin-related hereditary amyloid polyneuropathy (ATTR-PN)
Subcutaneous injection

Amyloid transthyretin cardiomyopathy (ATTR-CM)

Information

New molecular entity
AstraZeneca
Ionis Pharmaceuticals

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes

Category

Antisense drug designed to decrease the production of transthyretin to treat all forms of TTR amyloidosis. Antisense is a non-coding DNA strand of a gene - it is a way of silencing genes.
Amyloidosis is a hereditary, systemic disease due to TTR gene mutations. Mutations cause abnormal amyloid proteins to accumulate & damage body organs /tissues, causing intractable peripheral sensory and autonomic neuropathy, and/or cardiomyopathy. Familial amyloidosis caused by a transthyretin mutation occurs in ~1 in 100,000 Caucasians in the USA and ~4% in African Americans. Symptoms usually begin between 40 and 65 years of age.[1] Approx. 300,000- 500,000 patients worldwide have ATTR-CM [7].
Amyloid transthyretin cardiomyopathy (ATTR-CM)
Subcutaneous injection