The U.S. Food and Drug Administration has approved AstraZeneca‘s Calquence (acalabrutinib) for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.
The FDA’s accelerated approval, which allows for earlier approval of therapies that treat serious conditions and will fill a current unmet medical necessity, was based on the overall response rate seen in the ACE-LY-004 Phase 2 trial (NCT02213926). But continued approval of Calquence is dependent on further confirmation of the drug’s clinical benefit.
The approval came roughly two months after AstraZeneca submitted its new drug application. It was initially expected by March 2018.
“The accelerated approval of Calquence is a landmark moment for our company. It provides an exciting new treatment option for patients with mantle cell lymphoma and marks the first approval of a medicine that will be the cornerstone of our presence in hematology,” Pascal Soriot, chief executive officer of AstraZeneca, said in a press release. “Furthermore, today’s approval demonstrates our commitment to scientific leadership in Oncology and reinforces our progress towards returning to growth.”
The ACE-LY-004 study recruited 124 adult patients with relapsed or refractory MCL. Its primary objective was to determine patients’ overall response rate after treatment with Calquence, or how many patients experienced complete or partial shrinkage of their tumors after treatment.
The results showed that 80% of the patients responded to the treatment, including 40% who had a complete response and 40% who had a partial response.
The most common adverse events were anemia, low blood platelet levels, headache, low levels of immune cells called neutrophils, diarrhea, fatigue, muscle pain, and bruising. Due to these side effects, 1.6% of patients required a reduction in the treatment dose, and 6.5% discontinued the therapy.
This suggests that Calquence has a tolerable safety profile and may significantly impact the management of previously-treated MCL patients.
Full data from the ACE-LY-004 trial will be presented at an upcoming medical meeting, the company said. This will be the first time that AstraZeneca presents any acalabrutinib trial data.
Calquence is a highly selective and potent inhibitor of Bruton tyrosine kinase, a B-cell protein whose activity is abnormally increased in certain types of non-Hodgkin’s lymphoma, including MCL.
The drug is also being evaluated as a first-line therapy for MCL patients in the ACE-LY-308 Phase 3 clinical trial (NCT02972840).
“The acalabrutinib approval represents an important development for patients currently battling mantle cell lymphoma, an aggressive type of blood cancer that is typically diagnosed at an advanced stage and associated with a high relapse rate. In addition to the overall response rate, the high complete response rate of 40% seen in this trial illustrates the potential of acalabrutinib to help patients,” said Michael L. Wang, MD, professor, Department of Lymphoma/Myeloma, the University of Texas MD Anderson Cancer Center, and lead investigator of the ACE-LY-004 MCL clinical trial.