May 1, 2019 - Episode 1

FDA Approval in AML, Priority Review in Prostate Cancer, EMA CHMP Recommendations, and More


An FDA approval in acute myeloid leukemia, a priority review designation in prostate cancer, a new drug application in prostate cancer, and European recommendations in ovarian cancer, breast cancer, and cutaneous squamous cell carcinoma.

Welcome to OncLive News Network! I’m Gina Columbus.

The FDA has approved a supplemental new drug application for single-agent ivosidenib for the first-line treatment of adult patients with IDH1-mutant acute myeloid leukemia, as detected by an FDA-approved test, who are at least 75 years old or are ineligible to receive intensive chemotherapy.

The approval is based on findings from a phase I trial in which ivosidenib induced a 28.6% complete response rate and a CR plus CR with partial hematologic recovery rate of 42.9%. The median durations of CR and CR+CRh were not estimable, and 41.7% of patients who achieved CR/CRh remain on ivosidenib as of the data cutoff.

Results also showed that 58.3% of patients who achieved a CR or CRh were in remission at 1 year after receiving ivosidenib. The median time to CR/CRh was 2.8 months. Also, among 17 patients who were dependent on red blood cell and/or platelet transfusions at baseline, 41.2% were independent of RBC and such transfusions during any 56-day postbaseline period. Moreover, of the 11 patients who were independent of both RBC and platelet transfusions at baseline, 54.5% of them remained transfusion independent during the similar period.

The agency’s decision occurred more than a month before the action date, which was June 21, 2019.


The FDA has granted a priority review designation to a new drug application for darolutamide for use as a treatment for patients with nonmetastatic castration-resistant prostate cancer.

The NDA is based on data from the phase III ARAMIS trial, in which the investigational nonsteroidal androgen receptor antagonist was associated with a 59% reduction in the risk of metastases or death compared with placebo in this patient population.

Bayer, which is manufacturing darolutamide in collaboration with Orion Corporation, stated in a press release that darolutamide has also been granted fast track designation in this setting.

At a median follow-up of 17.9 months, results showed that the median MFS was 40.4 months with darolutamide versus 18.4 months in the placebo arm. Additionally, the MFS benefit was observed across patient subgroups, including those for baseline prostate-specific antigen doubling time, use of bone-targeting agents, Gleason score, age, and ECOG performance status.

The 3-year rates of OS were 83% in the darolutamide arm versus 73% with placebo, translating to a 29% reduction in the risk of death. Moreover, the median OS was not yet reached in either arm.


Also in prostate cancer, a supplemental new drug application has been submitted to the FDA for the approval of apalutamide for use as a treatment of patients with metastatic castration-sensitive prostate cancer.

The application is based on data from the phase III TITAN trial, which showed that apalutamide plus androgen deprivation therapy significantly improved radiographic progression-free survival and overall survival compared with placebo and ADT in this patient population. Full findings from the study will be presented at the 2019 ASCO Annual Meeting.

In January 2019, it was announced that due to these data from a preplanned analysis, along with a recommendation by an Independent Data Monitoring Committee, the study had been unblinded. Based on the positive results favoring apalutamide, it was recommended that patients on the placebo/ADT arm be permitted to cross over to receive apalutamide. Patients will continue to be followed for OS and long-term safety as part of the TITAN trial.

The sNDA is being reviewed as part of the FDA’s Real-Time Oncology Review program, which is designed to have a more efficient review process to make therapies more quickly available to patients.

The FDA initially approved apalutamide is currently indicated for the treatment of patients with nonmetastatic castration-resistant prostate cancer.


The European Medicines Agency’s Committee for Medicinal Products for Human Use made a handful of positive opinions this past week, recommending drugs for approval in breast cancer, ovarian cancer, and cutaneous squamous cell carcinoma.

First, the committee backed approval of talazoparib for adult patients with HER2-negative locally advanced or metastatic breast cancer harboring germline BRCA1/2 mutations. If approved, the PARP inhibitor’s indication would stipulate that eligible patients must have prior treatment with an anthracycline and/or a taxane in the neoadjuvant or adjuvant locally advanced or metastatic setting. Eligible patients with HR-positive disease also must first have received endocrine-based therapy.

The recommendation is based on data from the phase III EMBRACA trial, in which talazoparib reduced the risk of disease progression or death by 46% versus chemotherapy in patients with BRCA-positive advanced breast cancer.

In ovarian cancer, olaparib was recommended for approval as a frontline maintenance treatment for patients with BRCA-mutant advanced high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response following first-line platinum-based chemotherapy.

The positive opinion is based on findings from the phase III SOLO-1 trial, in which olaparib led to a 70% reduction in the risk of disease progression or death following response to platinum-based chemotherapy compared with placebo.

And in cutaneous squamous cell carcinoma, the committee issued a positive opinion recommending approval of cemiplimab for the treatment of adult patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not eligible for curative surgery or curative radiation.

The recommendation is based on a combined analysis of data from the phase II EMPOWER-CSCC-1 trial and 2 advanced CSCC expansion cohorts from a phase I trial of patients with metastatic and locally advanced CSCC.

Across the entire population, the overall response rate at a median follow-up of 8.9 months was 47%, the complete response rate was 4% and the partial response rate was 44%. The duration of response ranged from 1 month to over 15 months. Morever, 61% of patients had a duration of response of at least 6 months.


This week, we sat down with Dr Vicky Makker, of Memorial Sloan Kettering Cancer Center, to discuss evolving treatments for advanced endometrial cancer.

That’s all for today.

Thank you for watching OncLive News Network! I’m Gina Columbus.