Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted approval for a new indication for AKEEGA® (niraparib and abiraterone acetate dual-action tablet) plus prednisone. This decision marks a significant milestone, establishing AKEEGA as the first precision therapy specifically approved for the treatment of patients with BRCA2-mutated metastatic castration-sensitive prostate cancer (mCSPC).
This expanded approval addresses a critical unmet need for a patient population that typically faces a more aggressive form of the disease and a poorer prognosis. By combining a targeted therapy with an existing standard of care, AKEEGA offers a new, personalized approach to treatment.
The approval is based on compelling data from the Phase 3 AMPLITUDE study, a randomized, double-blind trial. In patients with BRCA2-mutated mCSPC, treatment with AKEEGA® plus prednisone and androgen deprivation therapy (ADT) significantly reduced the risk of radiographic progression or death by 54 percent compared to the current standard of care (placebo/abiraterone acetate plus prednisone and ADT). Furthermore, the combination prolonged the time to symptomatic progression by a notable 59 percent.
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There remains an urgent need for novel therapies for patients with BRCA2-mutated mCSPC, who face significantly faster disease progression and often shorter survival compared to those without the mutation,
AMPLITUDE is the first study to show that this precision medicine combination of a PARP inhibitor with an androgen receptor pathway inhibitor delays both radiographic and symptomatic disease progression.
Bradley McGregor
AKEEGA® is a dual-action tablet (DAT) that combines niraparib, a highly selective poly (ADP-ribose) polymerase (PARP) inhibitor, with abiraterone acetate, a CYP17 inhibitor. Patients are selected for therapy based on an FDA-approved test for genetic alterations.
The observed safety profile for the combination therapy was consistent with the known safety profile of its individual components. Johnson & Johnson also noted its commitment to patient access through the J&J withMe patient support program, which offers cost support and educational resources.
Detailed Efficacy and Trial Information
The FDA approval was primarily supported by the positive results from AMPLITUDE (NCT04497844), a Phase 3, randomized, double-blind, placebo-controlled, multicenter, global study.
- Study Population: The study enrolled 696 participants, including a subgroup with BRCA2 gene alterations.
- Primary Endpoint: The primary endpoint was radiographic progression-free survival (rPFS), which demonstrated the 54% reduction in the risk of progression or death.
- Mechanism of Action: AKEEGA is a dual-action tablet (DAT) combining two distinct classes of agents:
- Niraparib: A highly selective poly (ADP-ribose) polymerase (PARP) inhibitor. PARP inhibitors work by preventing cancer cells with damaged DNA-repair pathways (like those with BRCA2 mutations) from fixing themselves, leading to cell death.
- Abiraterone Acetate: A CYP17 inhibitor, which blocks the production of androgens (male hormones) that fuel prostate cancer growth.
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Safety and Adverse Reactions
The safety profile for AKEEGA® plus prednisone in the AMPLITUDE study was consistent with the known safety profile of its components.
- Most Common Adverse Reactions (occurring in $\geq20\%$ of patients): Decreased hemoglobin, decreased lymphocyte count, hypertension, decreased neutrophil count, musculoskeletal pain, decreased platelet count, constipation, fatigue, decreased potassium, increased creatinine, nausea, increased alkaline phosphatase, increased aspartate aminotransferase, respiratory tract infection, arrhythmia, increased blood bilirubin, and fluid retention/edema.
- Serious Adverse Reactions: Serious adverse reactions occurred in 36% of patients, with the most common being anemia ($\approx5\%$) and pneumonia ($\approx3.7\%$).
- Important Warnings: The therapy carries warnings and precautions related to:
- Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML)
- Myelosuppression (severe anemia, neutropenia, or thrombocytopenia)
- Hypokalemia, fluid retention, and cardiovascular adverse reactions
- Hepatotoxicity
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