On a roll, Boehringer wins 'breakthrough' status for pioneering leukemia drug

When Boehringer Ingelheim took volasertib into a Phase III study for acute myeloid leukemia earlier this year, the pharma company claimed a pioneering role in advancing a promising cancer therapy that inhibits polo-like kinase--or Plk. Today, the company said that the FDA agreed, offering to help speed development with its "breakthrough" therapy designation.

The BTD announcement marks another step forward in Boehringer's considerable effort to push a small group of late-stage cancer therapies into the market. In July the FDA approved afatinib for non-small cell lung cancer for patients with EGFR mutations. At ASCO last summer the company also reported positive--though slim--progression-free and overall survival data for nintedanib in a population of patients with advanced non-small cell lung cancer.

Now another late-stage oncology program has stepped into the spotlight. Boehringer says it was one of the first groups to push a Plk drug into the clinic. Volasertib inhibits Plk1, an enzyme that regulates cell division. By targeting the enzyme, the drug triggers apoptosis, blitzing cancer cells.

"This FDA Breakthrough Therapy designation provides Boehringer Ingelheim the opportunity to engage in an ongoing dialogue with the FDA to help expedite the development of volasertib as a potential treatment option for these patients with AML," said Sabine Luik, Boehringer's senior vice president of medicine and regulatory affairs.

Boehringer investigators barreled straight into Phase III in January after posting Phase II results on volasertib at the end of last year. Of the 42 patients who were ineligible for intensive remission induction therapy and treated with a combination of volasertib and LDAC, 13, or 31%, demonstrated a complete remission compared to 13% of the LDAC-only arm. The median event-free survival rate was 5.6 months in the combo group compared to 2.3 months in the chemo group.

All of Boehringer's cancer drugs are homegrown, stemming from a decision to start a cancer research division in Vienna 17 years ago. Now, that work is likely to bear fruit with some key approvals.

There's still quite a bit of discussion about what the FDA's breakthrough drug status actually means. The FDA's cancer chief, Richard Pazdur, made a full-throated effort at ASCO to pledge the agency's assistance to hastening a final marketing decision on cancer drugs ushered into the BTD program. But it's early days yet, with little hard evidence available to demonstrate just how meaningful it is for developers.

- here's the press release

Industry Voices: Breakthrough designation Q&A