Category: ATTR amyloidosis
Armed with new phase 3 data, AstraZeneca is preparing to file for regulatory approval of eplontersen, an antisense drug for one of the complications of the disease transthyretin amyloidosis (ATTR) it licensed from Ionis in a $3.6 billion deal last year. Eplontersen – previously known as IONIS-TTR-LRX – is designed to switch off the production […]
Four years after getting the first drug approved by the FDA to treat rare disease hereditary ATTR amyloidosis, Alnylam has got the go-ahead for a second – Amvuttra – which has a simpler, more patient-friendly dosing regimen. ATTR amyloidosis is an aggressive, rapidly progressing, debilitating and fatal disease, and the launch of Alnylam’s earlier Onpattro […]
AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of months. In the latest deal, the pharma group is paying $30 million upfront with another $730 million in milestone’s for global rights to Neurimmune’s NI006, […]
AstraZeneca has snapped up another Ionis-discovered drug for its pipeline, this time phase 3 candidate eplontersen for the disease transthyretin amyloidosis (ATTR). The deal is another sizeable one too, with an upfront fee of $200 million, another $485 million tied to regulatory approvals, and up to $2.9 billion in milestones depending on sales levels if […]
Alnylam has been a pioneer in hereditary ATTR amyloidosis, getting approval in 2018 for the first drug to treat the rare disease, and has now launched a digital companion to help patients track their symptoms. The new app – called STAR – allows patients to record their symptoms and share them with healthcare professionals to […]
Shots: The company has completed the enrolment in the P-III HELIOS-B study evaluates the efficacy and safety of vutrisiran (25 mg, SC, q3M, for ~ 36 mos.) vs PBO in 600 patients in a ratio (1:1) with ATTR amyloidosis with cardiomyopathy across 123 activated sites in 32 countries The 1EP of the study is to […]
Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis. The Danish drugmaker is paying $100 million upfront for rights to antibody drug PRX004, which is heading for phase 2 trials in cardiomyopathy associated with ATTR amyloidosis, a […]