UPDATED: Vertex's Kalydeco scores FDA panel rec in R117H. Are bigger nods next?

Despite falling short of its primary endpoint in a trial to examine its effects in cystic fibrosis patients with the R117H mutation, Vertex's ($VRTX) Kalydeco has scored an FDA advisory committee recommendation for approval in that population. And the way some see it, that's a sign of much, much bigger approvals to come.

On Tuesday, an agency panel voted 13-2 in favor of green-lighting the drug in R117H patients age 6 and older, the Boston-based company said in a statement. While the efficacy vote was closer in light of the trial miss--9 in favor, versus 6 against--Kalydeco still addresses a "high unmet medical need," Deutsche Bank analyst Robyn Karnauskas wrote in an investor note prior to the vote.

"We believe that doctors and patients under 18 years will want the drug," she said, as quoted by The Wall Street Journal's Pharmalot.

It's a good sign for Vertex, which could see a hefty chunk of new revenue if the FDA follows suit; according to the company, about 500 patients in the U.S. age 6 and older have the mutation, and with the drug boasting a $300,000 price tag, that can add up quickly.

But the way Sanford Bernstein analyst Geoff Porges sees it, is a "'soft indicator' for the agency's views of the next phase of Vertex's program," he wrote in an investor note seen by Pharmalot. The firm endorsement suggests "a generally positive view of the program, boding well for the approval of the much more important combination next year."

That "much more important" combo is Kalydeco plus lumacaftor, a duo that Vertex is testing in patients with the F508del mutation. According to ISI Group analyst Mark Schoenebaum, more than 28,000 patients worldwide are affected by F508del; industry watchers expect Vertex to file for that combo with the FDA later this year, he wrote in an investor note.

- read the release
- get more from Pharmalot

Special Reports: Top 15 Drug Launch Superstars - Kalydeco - Vertex Pharmaceuticals | Top 20 orphan drugs by 2018 - Kalydeco

Editor's note: The number of patients age 6 and older with the R117H mutation has been corrected to 500.

Suggested Articles

The future may be uncertain for AZ’s Imfinzi in first-line lung cancer, but its targeted med Tagrisso now boasts a green light in that setting.

Ultragenyx is back with another FDA nod, this time for Crysvita to treat X-linked hypophosphatemia in patients one year and older.

Roche got a two pieces of good Hemlibra news early this week—and what's good for Hemlibra must be good for Roche.