Clinical Management and Incidence of Thrombocytopenia and Glomerulonephritis in Patients with Hereditary ATTR Amyloidosis
Treated with Inotersen: Results from >5 Years of Clinical Trial and Post-Marketing Surveillance by Dr.
Patisiran, based on Nobel Prize-winning science, is an intravenously administered RNAi therapeutic targeting transthyretin for the treatment of hereditary ATTR amyloidosis
(6-8) The most common association is systemic AL amyloidosis, (9,10) but cases of diffuse alveolar-septal amyloidosis that are caused by systemic AA, systemic wild-type ATTR, and systemic hereditary ATTR amyloidosis
have also been reported (Table 2).
M2 PHARMA-October 1, 2018-Alnylam Submits New Drug Application in Japan for Onpattro for Treatment of Hereditary ATTR Amyloidosis
"This strategic restructuring enables streamlined development and an optimized approach to bringing innovative medicines to patients with ATTR amyloidosis
and hemophilia around the world, maximizing the commercial opportunities for these programs," said John Maraganore, Ph.D., chief executive officer of Alnylam.
The study involved the investigational RNAi therapeutic patisiran, which is being developed as a treatment for patients with hereditary ATTR amyloidosis
'We remain on track to report interim clinical data from the Phase 1 study of PRX004 for ATTR amyloidosis
in the fourth quarter of this year, and results from Part 1 of the PASADENA Phase 2 study of prasinezumab for Parkinson's disease are expected in 2020,' said Gene Kinney, Ph.D., President and Chief Executive Officer of Prothena.
Enrollment in our Phase 1 study of PRX004 in ATTR amyloidosis
is progressing and we now anticipate that the fourth quarter update on this program will include data from cohorts 1 through 4.
Alnylam Pharmaceuticals, a biopharmaceutical company, is halting the development of revusiran, a therapeutic intended for treating hereditary ATTR amyloidosis
with cardiomyopathy (hATTR-CM), it was reported yesterday.
We finished the quarter with over 500 patients on commercial therapy, and we expect continued and steady growth in the years to come driven by new patient finding, global expansion, and additional evidence generation in our ATTR amyloidosis
franchise,' said Barry Greene, President of Alnylam.
Akcea Therapeutics (AKCA) and Ionis Pharmaceuticals (IONS) announced new long-term, open-label extension, or OLE, data from the NEURO-TTR study of Tegsedi in patients with hereditary ATTR amyloidosis
, or hATTR, with polyneuropathy.
The product is intended for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis