dm+d

37504611000001104

New Medicines

Onpattro Hereditary transthyretin amyloidosis (ATTR) - progression after liver transplant

Information

Onpattro
Licence extension / variation
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Systemically delivered RNA interference therapy
Orthotopic liver transplantation (OLT) is used as a therapy for TTR amyloidosis. Survival after transplant may be associated with progression of neuropathy due to continued deposition of amyloid derived from wild-type TTR. More than 1,500 patients have had OLT for TTR amyloidosis. Data collected by the Familial Amyloidotic Polyneuropathy World Transplant Register show patients with the Val30Met mutation have a 5-year survival of 80%, in those with other mutations it is only 50-60% [1].
Hereditary transthyretin amyloidosis (ATTR) - progression after liver transplant
Oral

Onpattro Transthyretin amyloidosis (ATTR) with cardiomyopathy

Information

Onpattro
Licence extension / variation
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Systemically delivered RNA interference therapy
The clinical prevalence of wild-type ATTR cardiomyopathy is unknown, but autopsy studies suggest that up to 22-25% of indi- viduals >80 years old have demonstrable TTR amyloid deposits in cardiac tissue, although in most cases the degree of deposition is mild [1].
Transthyretin amyloidosis (ATTR) with cardiomyopathy
Oral