sNDA Submitted to FDA for Venetoclax Plus Acalabrutinib in Untreated CLL

Efficacy findings from the phase 3 trial published in the New England Journal of Medicine revealed that the combination elicited a 36-month PFS rate of 76.5% vs 66.5% with chemoimmunotherapy.

Developers have submitted a supplemental new drug application for a fixed duration, oral combination of venetoclax (Venclexta) in combination with acalabrutinib (Calquence) in patients with previously untreated chronic lymphocytic leukemia (CLL), according to a news release from AbbVie.1

Results from the phase 3 AMPLIFY trial (NCT03836261) form the basis of the submission, with the combination showing a progression-free survival (PFS) advantage vs chemoimmunotherapy. Efficacy findings from the phase 3 trial published in the New England Journal of Medicine revealed that the combination elicited a 36-month PFS rate of 76.5% (95% CI, 71.0%-81.1%) vs 66.5% (95% CI, 59.8%-72.3%) with chemoimmunotherapy (HR, 0.65; 95% CI, 0.49-0.87; P = .004).2

Additional efficacy results found that the estimated 36-month overall survival rates in the venetoclax and chemoimmunotherapy arms, respectively, were 94.1% (95% CI, 90.7%-96.3%) vs 85.9% (95% CI, 81.0%-89.6%), with an HR of 0.33 (95% CI, 0.18-0.56; P < .001). Furthermore, the 36-month event-free survival (EFS) rates were 75.9% vs 64.5%.

Response rates favored the venetoclax-based regimen, with an overall response rate (ORR) of 92.8% vs 75.2% with chemoimmunotherapy. Additionally, 28.1% of patients in the venetoclax arm had a response to treatment but subsequently experienced disease progression vs 33.9% in the chemoimmunotherapy group.

“This FDA submission marks a milestone for CLL treatment with the potential approval for the first oral combination regimen of [venetoclax] and acalabrutinib for [patients with] previously untreated chronic blood cancer. This new fixed-treatment duration approach could allow patients the opportunity for time off treatment, if approved, and be potentially practice-changing in frontline CLL care,” Svetlana Kobina, MD, vice president of Global Medical Affairs, Oncology at AbbVie, said in the news release.1

Patients with previously untreated CLL were randomly assigned 1:1:1 to receive venetoclax/acalabrutinib (n = 291), acalabrutinib/venetoclax/obinutuzumab (Gazyva; n = 286), or investigator’s choice of chemoimmunotherapy (n = 290). Crossover was not permitted on trial, and patients were stratified by age, IGHV mutational status, Rai stage, and region.

Those assigned to venetoclax/acalabrutinib received 100 mg of acalabrutinib twice daily from cycles 1 to 14 and venetoclax once daily from cycles 3 to 14, and a ramp-up dose over 5 weeks from 20 mg to 400 mg. Patients in the chemoimmunotherapy group received intravenous fludarabine/cyclophosphamide/rituximab (Rituxan) or bendamustine (Bendeka)/rituximab for the first 6 cycles according to standard dosing procedures. All treatments were given in 28-day cycles.

In the acalabrutinib/venetoclax and chemoimmunotherapy arms, the median age was 61 years (range, 31-84) and 61 years (range, 26-86), respectively. Most patients in either arm were male (61.2% vs 63.1%), treated in Europe (63.2% vs 63.1%), had an ECOG performance score of 0 or 1 (90.0% vs 90.3%), and unmutated IGHV (57.4% vs 59.3%). Additionally, 38.8% vs 42.8% of the respective arms had bulky disease greater than 5 cm, 47.1% vs 43.8% had Rai stage III or IV disease, and the median time from initial diagnosis to randomization in either arm was 28.5 months (IQR, 8.0-62.2) vs 29.6 months (IQR, 9.3-53.9).

The primary end point of the trial was PFS per blinded independent central review. Undetectable minimal residual disease was a key secondary end point. Other secondary end points included EFS, ORR, complete response rate with incomplete marrow recovery, and duration of response, as well as safety.

In the venetoclax/acalabrutinib and chemoimmunotherapy arms, respectively, 92.8% vs 91.1% of patients experienced any grade adverse effects (AEs). Grade 3 AEs occurred in 53.6% vs 60.6% of the respective arms, and serious AEs occurred in 24.7% vs 27.4%. Serious AEs leading to death occurred in 3.4% vs 3.5% of each arm, 2.7% vs 2.7% of which were related to COVID-19 infection.

The most frequent AEs of any-grade included neutropenia, hemorrhage, and COVID-19 infection in the acalabrutinib-venetoclax arm, and tumor lysis syndrome was only observed in 0.3% of this arm vs 3.1% of patients who received chemoimmunotherapy. No new safety signals were identified in the trials.

References

  1. AbbVie submits for U.S. FDA approval of combination treatment of VENCLEXTA® (venetoclax) and acalabrutinib for previously untreated patients with chronic lymphocytic leukemia (CLL). News release. AbbVie. July 29, 2025. Accessed July 29, 2025.
  2. Brown JR, Seymour JF, Jurczak W, et al. Fixed-duration acalabrutinib combinations in untreated chronic lymphocytic leukemia. N Engl J Med. 2025;392(8):748-762. doi:10.1056/NEJMoa2409804

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