BI leukaemia drug gains FDA breakthrough status
pharmafile | September 18, 2013 | News story | Research and Development, Sales and Marketing | Boehringer Ingelheim, FDA, acute myeloid leukaemia, breakthrough status
The FDA has granted Boehringer Ingelheim’s volasertib a ‘breakthrough status’ for patients with acute myeloid leukaemia.
Volasertib, a selective and potent polo-like kinase (Plk) inhibitor, is currently not licensed anywhere in the world, but this new status could speed up the drug’s approval in the US.
The breakthrough therapy designation is a recent initiative by the US regulator, intended for any drug that treats a serious or life-threatening condition and can show ‘substantial improvement’ over other drugs on the market.
The status was given by the FDA on the strength of a Phase II study in patients with previously untreated AML who were ineligible for intensive therapy.
The trial compared volasertib, in combination with the established chemotherapy of low-dose cytarabine (LDAC), versus LDAC alone.
The primary endpoint for the study was objective response (OR), responses of which were observed in 31% of patients (13 of 42 patients) treated with the combination of volasertib plus LDAC, compared to 13.3% of the patients (6 of 45 patients) treated with LDAC alone.
Secondary endpoints included the golden standard in oncology trials, overall survival (OS), which showed that BI’s drug and LDAC increased OS by eight months, compared to 5.2 months for LDAC alone.
“Volasertib’s innovative mode of action offers a new approach and may potentially provide a new therapy option for AML patients who have a high unmet medical need,” said Professor Klaus Dugi, corporate senior VP of medicine at Boehringer.
“We are looking forward to exploring opportunities with the FDA to expedite the final stage of the development of volasertib with the aim of making this treatment available for patients.”
If approved the drug will be competing with a number of established medicines for AML, including Celgene’s Vidaza and Janssen’s Dacogen.
There are also new drugs also in development, including Sunesis’ vosaroxin, Novartis’ PKC412 (midostaurin) and Cycacel’s sapacitabine.
But despite the crowding, this could be a lucrative market for those firms involved, as British analysts at GlobalData expect the acute myeloid leukaemia market to generate revenues of $430 million by 2017 – nearly three times its current value.
This is also good news for Boehringer as just last month it received US approval for its first oncology product, the late-stage lung cancer drug Gilotrif, which was also given an accelerated FDA approval. The privately owned German company will now hope that volasertib can be swiftly added to its growing oncology portfolio.
Leukaemia is a rare cancer of the bone marrow and blood and AML is one of the most common types of leukaemia in adults, accounting for around one-third of all adult leukaemias in the Western world.
AML has one of the lowest survival rates of all leukaemias and predominantly occurs in older adults, with the average age of newly diagnosed patients at 65 years old.
Ben Adams
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