STAT

State Prescription Drug Affordability Boards could end up limiting access to the life-saving cystic fibrosis medication Trikafta

I was living on the precipice of end-stage disease when I enrolled in the clinical trial that resulted in the historically fast Food and Drug Administration approval for the game-changing cystic ...

Sionna cystic fibrosis drug fails key trial, removing threat to Vertex

Sionna’s medicine didn’t have a meaningful additive impact when combined with Vertex’s top-selling Trikafta, surprising ...
FiercePharma

Vertex's Trikafta pricing central to cystic fibrosis treatment disparities, study says

The cost of a popular Vertex Pharmaceuticals therapy could be contributing to cystic fibrosis (CF) treatment disparities around the world, researchers argued in a recent paper. Just 12% of some ...
The Denver Post

$200,000-a-year cystic fibrosis medication deemed “affordable” by Colorado review board because of coupons

Getting your Trinity Audio player ready... Colorado’s new prescription drug review board decided in a first-in-the-nation vote that a medication costing more than $200,000 a year doesn’t qualify as ...

One Company’s Clinical Miss Is Another’s Moat: Vertex Rallies as Sionna’s CF Bet Collapses

Cystic fibrosis investors came to learn a harsh lesson on August 10 about how concentrated a market can be: Sionna Therapeutics, Inc. (NASDAQ:SION) shares fell more than 90% following a key trial ...

Sionna's SION-719 Failure Changes Everything (Rating Downgrade)

Sionna Therapeutics (SION) downgraded to Hold after SION-719 Phase 2a failure; cash-rich but pipeline risk rises.
FiercePharma

ICER rubber-stamps Vertex's $311K CF drug Trikafta but blasts 'monopoly' pricing

No one disputes the therapeutic value of Vertex Pharmaceuticals’ triplet drug Trikafta for cystic fibrosis—not even the cost watchdogs at the Institute for Clinical and Economic Review (ICER). But ...
Seeking Alpha

Vertex Pharmaceuticals: Cystic Fibrosis Leader With Lean Operating Model

The company has a 97% market share in the cystic fibrosis market with a forecasted 35% revenue growth in Trikafta drug in 2021. We expect its long-term growth to be driven by diversification to other ...

Sionna scraps genetic disorder drug program after trial setback; shares tank

Aug 10 (Reuters) - Sionna Therapeutics said on Monday it would stop developing its experimental drug as an addition to standard treatment for a type of genetic disorder after it failed to show a ...
nytimes.com

When a ‘Miracle’ Drug Is Out of Reach

A cystic fibrosis drug called Trikafta is widely available in the U.S. and Europe. It has transformed life for tens of thousands of people with the disease in high-income countries. But what happens ...
Nasdaq

Vertex Announces U.S. FDA Acceptance of Supplemental New Drug Application for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages ...

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has accepted its ...
Nasdaq

Vertex Announces FDA Approvals of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) for Use ...

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for ...