Tag:

cystic fibrosis

Latest Headlines

Latest Headlines

Vertex beefs up its cystic fibrosis pipeline with $1.2B Parion R&D pact

With its cystic fibrosis combo drug awaiting a likely approval at the FDA, Vertex is beefing up its pipeline in the field, paying $80 million upfront and promising up to $1.1 billion more in milestones to partner with Parion Sciences on a new class of CF-related drugs.

Vertex clears an FDA hurdle with its cystic fibrosis combo

A panel of FDA advisers voted in favor of Vertex Pharmaceuticals' closely watched combination treatment for cystic fibrosis, looking past some efficacy doubts and setting the stage for a likely approval.

FDA staffers raise doubts over key Vertex combo

As an FDA review nears for Vertex's Kalydeco-lumacaftor tandem--approval of which could help Kalydeco reach 10 times the number of cystic fibrosis patients it currently does--the agency's staffers have some concerns.

Spyryx Biosciences raises $18M with a novel take on cystic fibrosis

Vertex Pharmaceuticals has made a major difference--and a lot of money--in cystic fibrosis with its targeted treatments, drugs tailored for patients with particular genetic defects. But upstart Spyryx Biosciences believes it may have discovered a way to treat all forms of CF with a single therapy, and the company raised $18 million in Series A cash to advance the idea.

Yale collaborators use gene editing tech to fix a cystic fibrosis mutation

A project at Yale successfully adapted gene editing technology to correct the genetic mutation that is most commonly linked to cystic fibrosis.

Vertex gets 500-patient boost with FDA's newest Kalydeco nod

Vertex will start the new year with a group of new eligible patients for cystic fibrosis drug Kalydeco, thanks to a Monday green light from the FDA.

Is the CF Foundation's $3.3B royalty windfall good or bad for patients?

The Cystic Fibrosis Foundation announced today that it had cashed in its royalty stream from Vertex Pharmaceuticals' breakthrough CF drugs it backed for a $3.3 billion windfall. But where the foundation trumpeted the blockbuster deal with Royalty Pharma as a prelude to more investments into next-gen drugs, the New York Times ' Andrew Pollack had no trouble finding a critic eager to lambaste the foundation for the ultimate sin: putting profits ahead of patients.

CF biotech ProQR shoots for a $75M IPO to challenge Vertex

Dutch drug developer ProQR Therapeutics is swinging for a $75 million Wall Street debut to fund its work on a treatment for cystic fibrosis, looking to compete with market leader Vertex Pharmaceuticals.

Vertex scores 250 new Kalydeco patients with EMA approval

Vertex' patient pool for cystic fibrosis drug Kalydeco is getting wider--250 patients wider, to be specific. The European Medicines Agency has green-lighted the drug--originally approved for CF patients with at least one copy of the G551D mutation--for 8 additional mutations, spelling new revenue potential for the orphan drugmaker.

Arkansas stiff-arms CF patients seeking Vertex's $300K drug Kalydeco: WSJ

The drug-pricing battle continues, but this time, Gilead Sciences' Sovaldi isn't at the center.  That dubious honor goes to Vertex Pharmaceuticals' cystic fibrosis treatment Kalydeco. According to a lawsuit filed by three CF patients, Arkansas Medicaid officials are locking them out of Kalydeco treatment.