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.
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.
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.
A project at Yale successfully adapted gene editing technology to correct the genetic mutation that is most commonly linked to cystic fibrosis.
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.
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.
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' 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.
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.
Back in February, the FDA green-lighted Kalydeco to treat cystic fibrosis in patients with one of 8 distinct gating mutations, dramatically swelling the orphan drug's patient pool.