A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers ...

Health news and commentary gathered by MedPage Today staff ...

Following the announcement that NHS patients will be among first in Europe to benefit from access to ivacaftor, tezacaftor and elexacaftor, the appraisal of this triple therapy is now paused. NICE ...

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK ® (vanzacaftor ...

For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusivity – but there are signs ...

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) announced the U.S. Food and Drug Administration (FDA) has approved expanded use of ALYFTREK® (vanzacaftor/tezacaftor/ivacaftor) for the treatment of ...

Vertex Pharma has chosen the three-drug combination it intends to submit for approval later this year for cystic fibrosis, in a bid to add another product to its stable. The US company has plumped for ...

The agency has expanded the use of Alyftrek and Trikafta for treating patients with any genetic variant that results in production of CFTR proteins.

Introduction Since widespread availability of highly effective CFTRm elexacaftor/tezacaftor/ivacaftor, pregancy rates have doubled among fwCF in the UK. 1 ...

The expanded approval was based on clinical and/or in vitro data demonstrating responsiveness across 564 variants for Alyftrek and 521 variants for Trikafta. The label expansions were granted based on ...