Johnson & Johnson announced that the CHMP issued a positive opinion for AKEEGA®. It combines niraparib and abiraterone acetate for patients with metastatic hormone-sensitive prostate cancer and BRCA1/2 mutations. This recommendation marks a key step before European Commission approval and could expand treatment options for high-risk patients.
The CHMP’s endorsement followed strong Phase 3 AMPLITUDE study results. The trial compared niraparib plus abiraterone acetate with prednisone or prednisolone (AAP) against placebo plus AAP. Participants had metastatic hormone-sensitive prostate cancer and harmful homologous recombination repair (HRR) gene alterations. The study demonstrated significant improvements in radiographic progression-free survival compared with standard treatment.
Patients with BRCA1/2 mutations who received the niraparib/AAP combination did not reach median radiographic progression-free survival. In contrast, patients in the placebo plus AAP group progressed at 26 months. The therapy reduced the risk of radiographic progression or death by nearly 50%, providing long-term benefits for genetically defined patients.
The niraparib/AAP regimen also delayed symptomatic progression in patients with BRCA alterations. The study reported a 56% lower risk of symptom worsening, improving quality of life for patients with metastatic hormone-sensitive prostate cancer.
Early analysis suggested a trend toward improved overall survival with niraparib/AAP. Additional follow-up is needed to confirm these results. Researchers observed manageable side effects, including anemia and hypertension, consistent with expectations for each therapy.
Experts note that metastatic hormone-sensitive prostate cancer with BRCA1/2 mutations progresses faster and carries a poorer prognosis than non-mutated disease. These patients often advance quickly to metastatic castration-resistant prostate cancer, where treatment options are limited, and survival is shorter. Therefore, early targeted therapy represents a significant unmet medical need.
Henar Hevia, Ph.D., Senior Director and EMEA Therapeutic Area Head, Oncology, explained that patients with BRCA1/2-mutated prostate cancer historically had limited options. Pending approval, AKEEGA® could provide early intervention before resistance develops, improving patient outcomes.
The AMPLITUDE study enrolled 696 participants across 32 countries, making it one of the largest PARP inhibitor combination trials. Researchers conducted the trial as a randomized, double-blind, placebo-controlled, and international study. They presented their strong findings at major oncology conferences.
Research shows that about one in four patients with metastatic hormone-sensitive prostate cancer harbors homologous recombination repair gene alterations. BRCA1/2 mutations accelerate disease progression and shorten survival. This finding underscores the need for precision-targeted therapies.
Charles Drake, M.D., Ph.D., Vice President and Prostate Cancer Leader at Johnson & Johnson, said integrating targeted therapies into routine care represents a critical advance. He added that combining PARP inhibitors with hormonal therapy could complement standard care and reshape treatment for metastatic hormone-sensitive prostate cancer.
The current treatment commonly includes the use of androgen deprivation therapy (ADT), with or without other types of hormonal therapies. Many patients who started with this type of treatment developed resistance over time and then developed advanced metastasis and did not do well. The dual action mechanism of AKEEGA® is to block both hormone signal pathways and DNA repair pathways. Therefore, it provides specific treatment to genetically defined patients.
Safety data from AMPLITUDE showed adverse events were manageable with supportive care and dose adjustments. Despite some Grade 3/4 effects such as anemia and hypertension, discontinuations remained low, confirming tolerability.
The trial’s findings were featured at the 2025 American Society of Clinical Oncology Annual Meeting and included in the Best of ASCO program. These results highlighted strong clinical interest in this dual-action therapy.
Provided that the European Commission positively views the CHMP recommendation, niraparib plus abiraterone acetate, in conjunction with either prednisone or prednisolone, as well as ADT, will be available for prescription by clinicians who care for eligible patients. By providing an opportunity to expand precision oncology, this approval will ultimately lead to better methods of care for BRCA1/2-mutated metastatic hormone-sensitive prostate cancer patients.
Overall, CHMP endorsement demonstrates a continuing trend toward the use of genetically-targeted therapies. Enough clinical data support the recommended regimen for the two drugs with manageable safety concerns and an established pathway through the regulatory system showing promise that this two-prong strategy will provide additional options for patients with metastatic hormone-sensitive prostate cancer.
