A Look Back at FDA News from May 2019

In May 2019, the FDA approved several agents for the treatment of patients with acute myeloid leukemia (AML), breast cancer, hepatocellular carcinoma (HCC), renal cell carcinoma (RCC), chronic lymphocytic leukemia (CLL), and more.

In addition, the FDA granted several breakthrough therapy designations, fast track designations, and orphan drug designations. A partial clinical hold was also lifted in a clinical trial for cervical cancer, among other FDA news.

Here's a look back on the FDA happenings for the month of May 2019:

Ivosidenib Approved by FDA for Frontline IDH1+ AML

On May 2, 2019, ivosidenib (Tibsovo) monotherapy received FDA approval for the first-line treatment of adult patients with IDH1-mutant acute myeloid leukemia (AML), as detected by an FDA-approved test, who are ≥75 years old or are ineligible to receive intensive chemotherapy.

Adjuvant T-DM1 Approved by FDA for HER2+ Early Breast Cancer

The FDA approved ado-trastuzumab emtansine (T-DM1; Kadcyla) as an adjuvant treatment option for patients with HER2-positive early breast cancer who have residual invasive disease following neoadjuvant treatment with trastuzumab (Herceptin) and chemotherapy on May 3, 2019.

FDA Grants Fast Track Designation to Leronlimab for CCR5+ mTNBC

On May 7, 2019, the FDA has granted a fast track designation to a novel CCR5 antagonist, leronlimab (PRO 140), for use in combination with carboplatin to treat patients with CCR5-positive metastatic triple-negative breast cancer.

Ramucirumab Approved by FDA for Second-Line Treatment of AFP-Elevated HCC

On May 10, 2019, single-agent ramucirumab (Cyramza) received approval from the FDA for the treatment of patients with HCC who have an alpha fetoprotein of ≥400 ng/mL and have been previously treated with sorafenib (Nexavar).

Pomalidomide Receives FDA Breakthrough Designation for Kaposi Sarcoma

Pomalidomide (Pomalyst) was granted a breakthrough designation by the FDA on May 13, 2019, for use as treatment for both patients with HIV-positive Kaposi sarcoma who have had prior chemotherapy and patients with HIV-negative Kaposi sarcoma.

FDA Grants Orphan Drug Designation to P-BCMA-101 CAR Therapy for R/R Multiple Myeloma

On May 14, 2019, the FDA granted P-BCMA-101, an autologous chimeric antigen receptor (CAR) T cell, with an orphan drug designation for the treatment of patients with relapsed and/or refractory multiple myeloma.

Pexidartinib Supported by FDA's ODAC for Tenosynovial Giant Cell Tumors

A scheduled meeting of The FDA's Oncologic Drugs Advisory Committee (ODAC) supports the indication of pexidartinib as a treatment for adult patients with symptomatic tenosynovial giant cell tumor. On May 14, 2019, the panel announced they had voted 12 to 3 in favor of the CSF1R inhibitor; the FDA will now have to make its decision on the pexidartinib indication by the Prescription Drug User Fee Date of August 3, 2019. 

Novel Radiotherapeutic Granted FDA's Fast Track Designation for R/R Multiple Myeloma

Novel radiotherapeutic CLR 131 was granted a fast track designation from the FDA as a fourth-line or later treatment for patients with relapsed or refractory multiple myeloma on May 14, 2019.

FDA Panel Votes Not to Back Quizartinib Approval in AML

In an 8-3 vote on May 14, 2019, the FDA’s ODAC recommended against approving a new drug application (NDA) for quizartinib for adult patients with relapsed/refractory FLT3-ITD—positive AML. The FDA is now scheduled to make a final decision on the application by August 25, 2019.

Avelumab/Axitinib Approved by FDA for Frontline Treatment of RCC

On May 15, 2019, combination avelumab (Bavencio) plus axitinib (Inlyta) was approved by the FDA for the frontline treatment of patients with advanced RCC, based on findings from the phase III JAVELIN Renal 101 trial.

FDA Grants Novel; Agent Orphan Drug Designation for Adenoid Cystic Carcinoma

AL101 was granted an orphan drug designation by the FDA on May 15, 2019, for the treatment of patients with adenoid cystic carcinoma.

Venetoclax/Obinutuzumab Combo Approved by FDA for Frontline CLL

On May 15, 2019, the venetoclax (Venclexta) plus obinutuzumab (Gazyva) combination was approved by the FDA for the frontline treatment of patients with CLL or small lymphocytic lymphoma, based on findings from the phase III CLL14 trial.

FDA Lifts Partial Hold on AIM2CERV Trial of Investigational Immunotherapy Agent in Cervical Cancer

On May 15, 2019, the FDA lifted the partial clinical hold placed on the phase III AIM2CERV trial (NCT02853604), which is evaluating axalimogene filolisbac (AXAL) for the treatment of patients with high-risk locally advanced cervical cancer.

NovoTTF-100L Plus Chemotherapy Approved by FDA for Malignant Pleural Mesothelioma

The NovoTTF-100L System was approved by the FDA on May 23, 2029, in combination with pemetrexed and platinum-based chemotherapy as a frontline treatment for patients with unresectable, locally advanced or metastatic malignant pleural mesothelioma.

FDA Grants Novel TIL Therapy Breakthrough Therapy Designation for Advanced Cervical Cancer

The FDA granted a breakthrough therapy designation to novel tumor-infiltrating lymphocyte therapy LN-145 for the treatment of patients with recurrent, metastatic, or persistent cervical cancer who have progressed on or after chemotherapy on May 24, 2019.

Ruxolitinib Approved by FDA for Steroid-Refractory Acute GVHD

On May 24, 2019, ruxolitinib (Jakafi) received approval from the FDA as a treatment for adult and pediatric patients ≥12 years of age with steroid-refractory acute graft-versus-host disease.

Alpelisib Receives FDA Approval in Breast Cancer

Alpelisib (Piqray) was approved by the FDA on May 24, 2019, for the treatment of postmenopausal women, and men, with HR-positive, HER2-negative,PIK3CA-mutated, advanced or metastatic breast cancer following progression on or after an endocrine-based regimen based on data from the phase III SOLAR-1 trial.

Lenalidomide/Rituximab Receives FDA Approval for Indolent Non-Hodgkin Lymphoma

On May 28, 2019, the R²regimen of lenalidomide (Revlimid) plus rituximab (Rituxan) has been approved by the FDA for the treatment of patients with previously treated follicular lymphoma and marginal zone lymphoma (MZL), based on findings from the phase III AUGMENT trial.

FDA Grants Fast Track Designation to Lasofoxifene for ER+, ESR1-Mutant Metastatic Breast Cancer

The FDA granted a fast track designation on May 29, 2019, to the investigational agent lasofoxifene for the treatment of women with estrogen receptor (ER)—positive, HER2-negative metastatic breast cancer who have anESR1mutation.¹The agent is currently being investigated in a phase II trial in this setting.

ARV-110 Granted Fast Track Designation From FDA for mCRPC

On May 29, 2019, ARV-110, a PROTAC^®protein degrader, received a fast track designation from the FDA for the treatment of patients with metastatic castration-resistant prostate cancer who have disease progression following ≥2 systemic therapies.

FDA Approves Update to Gilteritinib AML Label to Include OS Data

A supplemental new drug application was approved on May 30, 2019, by the FDA to update the label for gilteritinib (Xospata) to include final analysis data from the phase III ADMIRAL trial, which demonstrated an improvement in overall survival with the FLT3 inhibitor compared with salvage chemotherapy in adult patients with relapsed/refractory FLT3-mutant AML.

Copanlisib Given FDA Breakthrough Designation for Marginal Zone Lymphoma

On May 30, 2019, copanlisib (Aliqopa) has been granted with a breakthrough therapy designation from the FDA for the treatment of adult patients with relapsed MZL who have received at least 2 prior therapies.

Tazemetostat Submitted for FDA Approval in Epithelioid Sarcoma

An NDA for tazemetostat was submitted to the FDA for the treatment of patients with epithelioid sarcoma who are not eligible for curative surgery on May 30, 2019.

FDA Grants Priority Review to Daratumumab/VTD Combo in Frontline Multiple Myeloma

On May 31, 2019, the FDA granted a priority review designation to a supplemental Biologics License Application for daratumumab (Darzalex) in combination with bortezomib (Velcade), thalidomide (Thalomid), and dexamethasone (VTd) for the treatment of newly diagnosed patients with multiple myeloma who are candidates for autologous stem cell transplant.

HER2-Targeted Antibody ZW25 Earns FDA Fast Track Designation in GEA