Another FDA Approval For Vertex (VRTX)

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) has announced another FDA approval.  While this approval will treat cystic fibrosis, the current total patient population is rather small if the numbers are not meant to be much more expanded than what is indicated. Still, the initial stock pop has been rather impressive considering the commentary.

The U.S. Food and Drug Administration has approved its KALYDECOTM as the first medicine to treat the underlying cause of cystic fibrosis.  KALYDECO is approved for people with CF ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator gene.

Here is where we are concerned over the bump: “Approximately 1,200 people in the United States, or 4 percent of those with CF, are believed to have this mutation.”

The good news is that this drug KALYDECO was granted a very rapid approval… just 3 months.  The company called it “one of the fastest FDA approvals ever and marking the second approval of a new medicine from Vertex in less than a year.”

Vertex has also noted that it has established a financial assistance and patient support program to help get KALYDECO to eligible patients for whom it is prescribed.

KALYDECO was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

Vertex shares are up almost 8% at $37.60 on more than 5 million shares and the market cap is now about $7.8 billion.  Its 52-week trading range is $26.50 to $58.87.

UPDATE: Vertex shares are currently halted

JON C. OGG