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In Case You Missed It!
Week of July 31 - August 04

Cranbury, NJ—This week, the U.S. Food & Drug Administration (FDA) has announced multiple new approvals or indications in the fields of hematology and oncology.

1. Nivolumab granted accelerated approval for MSI-h/dMMR colorectal cancer

On Monday, July 31, the FDA granted accelerated approval for the PD-1 inhibitor, nivolumab, in patients with mismatch repair deficient (dMMR), microsatellite instability high (MSI-H), metastatic colorectal cancer that has progressed following chemotherapy-based treatment. This approval was based off of data from the CHECKMATE 142 trial (NCT02060188). The multicenter, open-label, single arm phase II study enrolled 53 patients. Patients received nivolumab 3 mg/kg via intravenous infusion every 2 weeks. An overall response rate (ORR) of 28% was observed. Duration of response was ≥6 months in 67% of patients. Nivolumab has previously been approved for use in multiple disease types including melanoma, lung cancer, renal cell carcinoma. A full release and prescribing information for nivolumab can be found on the FDA’s website.

2. Enasidenib approved for IDH2-mutated RR-AML

On Tuesday, August 01, the FDA approved an isocitrate dehydrogenase-2 (IDH2) inhibitor, enasidenib, in adult patients with relapsed or refractory acute myeloid leukemia (AML) with an IDH2 mutation. Enasidenib has been approved for use with a companion diagnostic, RealTime IDH2 Assay. This approval is based off results from a multicenter, open-label, single arm phase I/II trial (NCT01915498) enrolled 199 patients with relapsed or refractory AML. After a minimum of 6 months of treatment, 19% of patients achieved complete remission (CR) for a median of 8.2 months, an additional 4% of patients achieved complete remission with partial hematologic recovery (CRh). Of the 157 patients who required transfusions due to AML, 34% were transfusion independent following treatment with enasidenib.


“[Enasidenib] is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH2 mutation,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “The use of [enasidenib] was associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”

3. Ibrutinib granted expanded approval for cGVHD

On Wednesday, August 02, the FDA expanded the approval of ibrutinib, a small molecule oral inhibitor of Bruton’s tyrosine kinase (BTK), for the treatment of adults with chronic graft versus host disease (cGVHD) following hematopoietic stem cell transplantation (HSCT). The supplemental new drug application is based on data from the multicenter, open label, single-arm phase Ib/II PCYC-1129 trial (NCT02195869). In the study, an ORR of 67% was observed in patients who did not respond to at least 1 prior line of treatment for cGVHD, 21% of responders had a CR, and 45% of patients had a partial response.

“Patients with cGVHD who do not respond to other forms of therapy—typically corticosteroids to suppress their immune system—now have a treatment option specifically indicated to treat their condition,” said Pazdur in a press release. “This approval highlights how a known treatment for cancer is finding a new use in treating a serious and life-threatening condition that may occur in patients with blood cancer who receive a stem cell transplant.”

4. New chemotherapy combination approved for certain types of AML

Then, on Thursday, August 03, the FDA approved CPX-351 for treatment of adult patients with newly diagnosed therapy-related AML (t-AML) and AML with myelodysplasia-related changes (AML-MRC). This approval is based off of multicenter, randomized phase III trial (NCT01696084) evaluating CPX-351 versus standard 7+3 therapy. Patients receiving CPX-351, a liposomal bound formulation of cytarabine and daunorubicin in a 5:1 molar ratio, had a median overall survival (OS) of 9.56 months, while 7+3 was associated with a median OS of 5.95 months, a 31% reduction in risk of death.

“This is the first approved treatment specifically for patients with certain types of high-risk AML,” said Pazdur in a press release. “[CPX-351] combines two commonly used chemotherapies into a single formulation that may help some patients live longer than if they were to receive the two therapies separately.” This approval was based off of data presented at the 2016 ASCO Annual Meeting.


Published Online: Friday, August 04, 2017

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