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news.The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) ha adopted three positive opinions recommending orphan drug designation to Acerta Pharma's acalabrutinib (ACP-196) cancer drug.
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Last December, AstraZeneca agreed to acquire a 55% stake in privately-owned biotech firm Acerta Pharma for $4bn.
Acalabrutinib has been recommended as an orphan product for chronic lymphocytic leukaemia or small lymphocytic lymphoma, mantle cell lymphoma and lymphoplasmacytic lymphoma.
The drug is a highly selective, irreversible, second generation Bruton’s tyrosine kinase (BTK) inhibitor, with about 1,000 patients treated so far in clinical trials throughout development program.
More than 600 patients have been treated with acalabrutinib monotherapy. Phase I/II data demonstrated a favourable safety profile and efficacy in relapsed/refractory chronic lymphocytic leukaemia patients.
AstraZeneca executive vice-president of global medicines development and chief medical officer Sean Bohen said: "Today’s three positive opinions recommending acalabrutinib for designation as an orphan medicinal product are important milestones.
"The positive opinions underscore the continued need for the development of new therapies in these serious and life-threatening conditions and support our commitment to bring new medicines to patients as quickly as possible."
Acalabrutinib is also being tested in several phase I/II studies in solid tumours, as monotherapy or in combination with immune checkpoint inhibitors or other standard of care regimens.