UPDATED: Novartis grabs FDA's coveted 'breakthrough' status for lung cancer drug

The FDA has opened the inside track to Novartis' experimental lung cancer drug, which gained a "Breakthrough Therapy" designation that speeds the development and review schedules for new treatments. The Swiss drug giant ($NVS) plans to file for approval the drug, now in mid-stage clinical trials, in early 2014. Since clinical development began in 2011, the program has advanced with lightning speed compared with those that take 10 years or so to trial before submitted for approval. 

While there are no guarantees of an FDA approval for Novartis' compound, code-named LDK378, the "breakthrough" tag provides an early nod to the potential of the candidate to improve treatment for patients with metastatic non-small cell lung cancer with anaplastic lymphoma kinase (ALK) mutations. The Novartis compound could offer another option for these patients whose cancer progresses despite treatment with Pfizer's ($PFE) approved drug Xalkori (crizotinib).

The "breakthrough" designation is also important because Novartis' compound and others with the coveted status have a shot to be approved by the FDA without completing all three phases of clinical trials typically required before an approval decision. The FDA began awarding the new status this year to promising treatments, including Vertex's ($VRTX) Kalydeco and VX-809 for cystic fibrosis and the experimental lymphoma drug ibrutinib from Pharmacyclics ($PCYC) and its partner Johnson & Johnson ($JNJ).

Novartis' LDK378 joined the "breakthrough" club after showing an 80% response rate in patients studied in Phase I trial of 88 subjects with advanced cases of ALK-positive NSCLC. The company has already begun a pair of Phase II studies of the compound for patients with the same kind of ALK-positive cancers, which account for about 3% to 8% of cases of NSCLC. And plans call for kicking off Phase III development of the new drug later this year.  

"LDK378 is a strong example of our research approach, which focuses on identifying the underlying cause of disease pathways," said Alessandro Riva, Novartis' global head of oncology development, in a statement. "This Breakthrough Therapy designation will allow us to collaborate more closely with the FDA and potentially to expedite the availability of an important new treatment option for patients with ALK+ NSCLC."

Despite the percentage of NSCLC patients with ALK mutations, pharma outfits are advancing multiple new drugs for this population. Those candidates include Ariad Pharmaceuticals' ($ARIA) AP26113 and TSR-011, which developer Tesaro ($TSRO) licensed in from biotech giant Amgen ($AMGN). Both those two compounds are in Phase I trials.

- here is Novartis' release

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