Roche/AbbVie's venetoclax grabs third FDA 'breakthrough' for blood cancer

By Ben Adams

AbbVie ($ABBV) and Roche's ($RHHBY) venetoclax has received yet another accelerated review from the FDA--this time in patients with the most common type of acute leukemia.

Specifically, the regulator has given the North Chicago, IL, drugmaker and its Swiss partner a third Breakthrough Therapy Designation (BTD) for the drug, in combination with hypomethylating agents, for the treatment of patients with first-line acute myeloid leukemia (AML) who can't receive high-dose chemotherapy.

Venetoclax (ABT-199) works as an inhibitor of the B-cell lymphoma-2 protein and is being developed by AbbVie in partnership with cancer drug specialist Roche/Genentech.

Both companies are seeking to offset looming biosimilar threats in the near future with new biologic approvals, with AbbVie in particular seeking to wean itself off its aging $13-billion-a-year autoinflammatory drug Humira (adalimumab) before it loses its patents.

It's unusual for one drug to gain three separate BTDs--which speed up the time it takes the FDA to assess a medicine--but it will likely be a boon for the drug's financial prospects. Analysts are predicting sales of $2 billion for the drug by the end of the decade should it gain its approvals.

This latest priority review adds to the decision in April last year to allow a BTD for venetoclax in previously treated patients with chronic lymphocytic leukemia (CLL) with the 17p deletion genetic mutation.

Earlier this month, AbbVie also announced that the FDA had granted another BTD for the medicine in the treatment of patients with relapsed/refractory CLL, in combination with Roche's established blood cancer drug Rituxan (rituximab).

AbbVie CSO Michael Severino

Dr. Michael Severino, executive vice president of R&D and chief scientific officer at AbbVie, said: "This third Breakthrough Therapy Designation for venetoclax highlights our efforts to pursue the significant and broad potential of this therapy."

Currently, no single standard of care exists for older patients with AML, as there are no FDA-approved therapies for patients not able to receive standard induction therapy or intensive chemotherapy.

However, privately owned Boehringer Ingelheim is currently studying its AML drug volasertib, an inhibitor of polo-like kinase (Plk), in Phase III trials and was awarded a BTD in the U.S. in 2013 in the same therapy setting as venetoclax.

And Agios ($AGIO) and Celgene ($CELG) are also working through a Phase III AML study with its top prospect AG-221, with results expected next year.

More than 20,000 people were diagnosed with AML in the U.S. in 2015, making it the most common type of acute leukemia among adults.

- here's the release

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