Category: ATTR amyloidosis

  • AZ preps FDA filing for $3.6bn amyloidosis drug eplontersen

    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 […]

  • Alnylam grabs FDA approval for its second amyloidosis therapy

    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 goes bigger in amyloidosis with Neurimmune deal

    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, […]

  • AZ gets closer to Ionis, licensing amyloidosis drug in $3.6bn deal

    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 launches digital tool for AATR amyloidosis patients

    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 […]

  • Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

    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 […]