AstraZeneca and Acerta Pharma announced Thursday the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) has adopted three positive opinions recommending the cancer drug acalabrutinib for the orphan medicinal product designation in three indications.
Orphan status is awarded to medicines promising significant benefit in the treatment of rare, life-threatening diseases and the designation provides companies with special development and market exclusivity incentives.
The three positive opinions of acalabrutinib (ACP-196) are for the treatment of chronic lymphocytic leukaemia or small lymphocytic lymphoma, mantle cell lymphoma and lymphoplasmacytic lymphoma.
Sean Bohen, executive vice-president of Global Medicines Development and chief medical officer at AstraZeneca, said in a statement: "Today's three positive opinions recommending acalabrutinib for designation as an orphan medicinal product are important milestones. They reinforce the strategic rationale for our investment in Acerta, demonstrating clear progress in developing a potential best-in-class medicine that could transform treatment for patients across a range of blood cancers."
The drug is currently in the final stages of clinical development for various blood cancers, apart from the three indications.
The drug is a Bruton's tyrosine kinase (BTK) inhibitor with a mode of action similar to AbbVie and Johnson & Johnson's blood cancer drug Imbruvica (ibrutinib), targeting a range of blood cancers and solid tumours.
In December 2015, AstraZeneca had announced it was buying 55 percent stake in privately-held biotech firm Acerta Pharma for about $4 billion, Reuters reported. AstraZeneca believes that acalabrutinib can generate sales of more than $5 billion a year.
Reuters had also reported once acalabrutinib was approved in the United States and Europe, Acerta shareholders would have the option to sell the remaining 45 percent of shares in the biotech company to AstraZeneca for approximately $3 billion.