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.
Any drugmaker knows that no matter what a drug's sticker price, its sales can only go as far as its patient pool will take it. So for Vertex, whose cystic fibrosis med Kalydeco had reached nearly all eligible patients in the U.S. and Europe, a new FDA approval to treat more CF sufferers is pretty significant.
Vertex Pharmaceuticals' piecemeal approach to the world's cystic fibrosis sufferers took a hit as Kalydeco failed to meet its primary endpoint in a Phase III trial on patients with one form of the disease, sending the company's shares down as much as 6% premarket on Thursday morning before an eventual rebound.
What happened? A new wave of hepatitis C treatments, led by Gilead Sciences' sofosbuvir. Heartily backed by an FDA advisory panel Friday, sofosbuvir is first in a class of treatments aimed at shortening hep C treatment, boosting its effectiveness and easing its notorious side effects. The coming all-oral drug cocktails will shut out interferon--and shunt Incivek aside.