FDA Updates: Approvals and Designations in Leukemia, Prostate Cancer, and Melanoma
核心洞察
Capivasertib plus abiraterone and ADT shows improved radiographic progression-free survival in PTEN (搜索)-deficient metastatic hormone-sensitive prostate cancer (搜索).
LBS-007 receives FDA fast track designation for acute myeloid leukemia (搜索) treatment based on promising early response data from a phase 1/2 trial.
BLA submitted for RP1 with nivolumab for advanced melanoma (搜索) patients who progressed on prior PD-1 inhibitor therapy.
The FDA has been active in recent weeks, with several key decisions and designations impacting the treatment landscape for various cancers. These include approvals, designations, and application acceptances that promise to bring new options to patients and clinicians.
Capivasertib Combination in Prostate Cancer
Capivasertib (Truqap), when combined with abiraterone acetate (Zytiga) and androgen deprivation therapy (ADT), has demonstrated a significant improvement in radiographic progression-free survival for patients with PTEN (搜索)-deficient de novo metastatic hormone-sensitive prostate cancer (搜索) (mHSPC). These findings come from the phase 3 CAPItello-281 trial (NCT04493853), where the combination was compared against placebo plus abiraterone and ADT. While overall survival (OS) data were still immature at the time of analysis, a trend favoring the capivasertib regimen was observed.
Susan Galbraith, executive vice president of Oncology research and development at AstraZeneca, noted, "These results show for the first time that adding an AKT inhibitor to a standard-of-care therapy can provide benefit to patients with a biomarker of PTEN (搜索)-deficient mHSPC." She emphasized the potential role of this combination in an area of critical unmet need.
Fast Track Designation for LBS-007 in AML
LBS-007 has been granted fast track designation by the FDA for the treatment of patients with acute myeloid leukemia (搜索). This decision is supported by data from a phase 1/2 trial (NCT05756322) evaluating LBS-007 in patients with relapsed or resistant acute leukemias. The phase 1 portion of the study is assessing LBS-007 both as a monotherapy and in combination with venetoclax (Venclexta) and azacitidine. Phase 2 will further evaluate the agent as a monotherapy and in combination therapy at the dose identified in phase 1.
Tom Lin, chairman of Lin BioScience (搜索), expressed enthusiasm about the early treatment responses seen with LBS-007, highlighting its potential efficacy in addressing a critical unmet medical need.
BLA Submission for RP1 in Melanoma
A biologics license application (BLA) for RP1 (vusolimogene oderparepvec) in combination with nivolumab (Opdivo) has been submitted to the FDA. The application seeks approval for patients with advanced melanoma (搜索) who have previously been treated with a PD-1 inhibitor-containing regimen. The submission is based on data from the phase 1/2 IGNYTE trial (NCT03767348), an open-label, dose-escalation and -expansion study evaluating safety and efficacy.
Additionally, the confirmatory phase 3 IGNYTE-3 trial (NCT06264180) is currently enrolling patients to assess RP1 in combination with nivolumab. Eligible patients include those with advanced melanoma (搜索) who have progressed on anti–PD–1 and anti–CTLA-4 therapy or who are not candidates for anti–CTLA-4 treatment.
Sushil Patel, CEO of Replimune, stated, "Today is an important milestone for Replimune and for the melanoma (搜索) community as we are one step closer to having another potential treatment available for patients who have limited options after progressing on anti–PD-1–containing regimens."
FDA Approves Revumenib for KMT2A-Rearranged Acute Leukemia
Revumenib (SNDX-5613) has received FDA approval for the treatment of adult and pediatric patients with relapsed/refractory (R/R) KMT2A (搜索)-rearranged acute leukemia (搜索). This approval is based on data from the phase 1/2 AUGMENT-101 trial (NCT04065399), which evaluated the efficacy and safety of the drug in 94 patients with R/R KMT2Ar acute leukemia, acute lymphoblastic leukemia (ALL)/mixed phenotype acute leukemia, and acute myeloid leukemia (搜索) (AML).
Eytan M. Stein, MD, from Memorial Sloan Kettering Cancer Center, commented, "KMT2A (搜索)-rearrangements occur in patients with acute myeloid and ALL, in adults, children, and overall, and have a poor prognosis. Revumenib gives hope to those with relapsed and refractory KMT2Ar disease."