Anavex Life Sciences Corp (AVXL)

[XenaLives]

Gee fixing misfolded proteins may have a lot of applications, who'd a thunk it?

UPDATED: Pfizer enters Alnylam, Ionis’ TTR turf with early approval for cardiomyopathy drug, carrying a $225K price tag

by Natalie Grover

— on May 6, 2019 09:10 AM EDT
Updated: 09:20 AM


Pfizer is set to pull its weight in transthyretin-mediated amyloidosis, a field that has so far been Alnylam and Ionis’ turf.

Transthyretin (TTR) is a relatively abundant protein in the blood, which transports thyroxine and retinol. But it can become unstable and misfold — due to a genetic mutation or environmental factors — sparking an accumulation of toxic amyloid aggregates in the heart or peripheral nerves.

On Monday, the big US drugmaker $PFE said the FDA had approved two oral formulations of its transthyretin-stabilizer tafamidis — Vyndaqel (80 mg, taken as four 20mg capsules) and Vyndamax (61 mg, taken as a once-daily single capsule) — to treat cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults, to reduce related hospitalization and death.

https://endpts.com/pfizer-enters-alnylam-ionis-ttr-turf-with-early-approval-for-cardiomyopathy-drug-carrying-a-225k-price-tag/