- The European Medicines Agency (EMA) has recommended the cancer drug acalabrutinib receive orphan drug status, giving Acerta Pharma a major boost. AstraZeneca holds a 55% stake in Acerta from a $4 billion deal in December 2015.
- Acalabrutinib is a Bruton's tyrosine kinase inhibitor and is designed to treat chronic lymphocytic leukaemia or several types of lymphoma.
- The drug is in phase 3 trials for B-cell blood cancers, as well as phase 1/2 trials for multiple solid tumors.
AZ bought a controlling stake Acerta mainly to gain rights to acalabrutinib. AZ sees mega-blockbuster potential with this late-stage candidate, with estimates of $5 billion per year. If acalabrutinib makes it to market, it will be competing directly with Johnson & Johnson's Imbruvica. However, AZ has suggested acalabrutinib may offer superior efficacy and a better side-effect profile, reports Reuters.
In addition to treating blood cancer, acalabrutinib may also have potential for treating solid tumors and certain autoimmune diseases.
If acalabrutinib is approved in the U.S. and the E.U., Acerta shareholders will have the option to sell the remaining 45% of the company to AZ for an additional $3 billion.