Novartis races to the FDA with its 'breakthrough' lung cancer drug

Novartis ($NVS) isn't wasting any time with LDK378, its promising in-development lung cancer drug, filing for FDA approval before embarking on a Phase III study for the breakthrough-designated therapy.

With the application, Novartis is swinging for the full potential of the FDA's newfangled breakthrough status, under which the agency can approve drugs based on mid-stage data. And that's just what the company has in mind, believing its non-small cell lung cancer treatment can sway regulators based on Phase II results alone.

The treatment, designed for patients with metastatic non-small cell lung cancer with anaplastic lymphoma kinase mutations, picked up the FDA's coveted breakthrough therapy designation last year after showing an 80% response rate in a Phase I trial on 88 patients with advanced cases of the disease.

The FDA has adopted 37 drugs into its breakthrough club since launching the program in 2012, but of the three therapies since approved--Roche's ($RHHBY) Gazyva, Johnson & Johnson ($JNJ) and Pharmacyclics' ($PCYC) Imbruvica, and Gilead's ($GILD) Sovaldi--not one made it to the market without a Phase III study.

While the early submission underlines the ambition of Novartis' oncology wing--which expects to launch 10 of its pipeline drugs by 2017--it could well undercut Ariad Pharmaceuticals' ($ARIA) comeback story. The beleaguered biotech finally got its breadwinning Iclusig back on the market, but Novartis' rapid progress with LDK378 might spell trouble for Ariad's similar AP26113, for which the company failed to secure a breakthrough tag last year. Ariad's shares fell about 3% on the news but are still up nearly 20% on the strength of some widely questioned buyout rumors.

LDK378 is among three Novartis drugs granted breakthrough status last year, joining the orphan drug BYM338 and the heart failure-treating serelaxin.

- read the statement

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