Novartis promises to test the full value of FDA's 'breakthrough' status

Ever since the FDA started to anoint a short list of drug candidates as "breakthrough" therapies deserving of swift regulatory reviews, the industry has been trying to puzzle out one important question: What does it mean?

The promise that the FDA would view these therapies' progress with a sense of urgency offers a chance to get fast feedback. But would the time it takes to progress one of these drugs through the clinic be significantly shortened, perhaps allowing biopharma companies a chance to get an approval right after the Phase II mark?

Novartis ($NVS) is about to put the proposition to a test. In discussions with Bloomberg, its development team says they could file for an approval of the breakthrough drug LDK378 after Phase II, which is typically the time when developers prep a lengthy late-stage effort that can prove a new cancer drug can prolong lives--the overall survival gold standard that has long dominated development cycles in oncology. That would put a new drug application at the FDA in early 2014.

Herve Hoppenot, Novartis's president of oncology, tells Bloomberg that a successful drug application could make LDK378 available after just three years of testing, one of the fastest time-to-market records they've ever seen.

Novartis has recorded some promising early-stage data on the drug. The therapy reduced the size of tumors in four of five patients, the kind of response that could indicate that patients with tough cases of lung cancer would live longer if this therapy was available. More data are expected at the upcoming ASCO meeting in Chicago.

"At the end of the day, this is about giving in to patients' demand for faster access," Helvea analyst Olav Zilian told Bloomberg. "The proof that the drug works will have to be handed in later."

Like many of its Big Pharma rivals, Novartis has plenty to worry about on the revenue front as generic competition looms. But the one revenue projection for LDK378, $360 million a year, would do little to fill a multibillion-dollar gap in sales. Still, Novartis is known for making short but fast development steps as it breaks into categories and then starts to build sales. That could make Novartis one of the ideal industry candidates to show just what "breakthrough" status is worth.

- here's the story from Bloomberg