A Look Back at the FDA News from September 2020

Here is a look back at the FDA happenings from the month of September 2020.

During the month of September 2020, the FDA granted 2 new approvals for the treatment of patients with acute myeloid leukemia and a subset of patients with lung cancer. There were also 3 new Fast Track designations, 3 Breakthrough Therapy designations, 3 Priority Review designations, and 3 Orphan Drug Designations.

Here is a look back at the FDA happenings from the month of September 2020:

FDA Approves CC-486 as Maintenance Therapy in Acute Myeloid Leukemia

On September 1, 2020, the FDA approved the oral hypomethylating agent CC-486 (azacitadine tablets, Onureg) as maintenance for adult patients with acute myeloid leukemia who achieved a first complete remission or with incomplete blood count recovery after intensive induction chemotherapy and who are unable to complete intensive curative therapy.

FDA Grants Orphan Drug Designation to RNL in Recurrent Glioblastoma

The FDA granted an Orphan Drug designation on September 1, 2020, to rhenium nanoLiposomes for the treatment of patients with recurrent glioblastoma.

FDA to Review sBLA for Axi-Cel in R/R Follicular Lymphoma and Marginal Zone Lymphoma

The FDA accepted a supplemental Biologics License Application on September 4, 2020, for axicabtagene ciloleucel (axi-cel; Yescarta) for the treatment of patients with relapsed or refractory follicular lymphoma or marginal zone lymphoma after 2 or more prior lines of systemic therapy.

FDA Approves Pralsetinib in RET Fusion-Positive Non-Small Cell Lung Cancer

The FDA approved pralsetinib (formerly BLU-667, Gavreto) for the treatment of adult patients with metastatic RET fusion–positive non–small cell lung cancer on September 4, 2020.

FDA Grants Priority Review to Pralsetinib for RET+ Thyroid Cancers

On September 4, 2020, the FDA granted a priority review to pralsetinib (formerly BLU-667; Gavreto) for the treatment of patients with advanced or metastatic RET-mutant medullary thyroid cancer as well as for patients with RET fusion–positive thyroid cancer.

FDA Grants Orphan Drug Designation to Novel Bcl-1 Inhibitor in CLL

A novel Bcl-2 inhibitor, APG-2575, been granted an Orphan Drug designation (ODD) by the FDA for the treatment of patients with chronic lymphocytic leukemia on September 7, 2020.

FDA Grants Orphan Drug to Bispecific Antibody as Treatment of Thymic Epithelial Tumors

The FDA granted an Orphan Drug designation on September 3, 2020, to KNO46 for the treatment of thymic epithelial tumors, Alphamab Oncology, developer of this recombinant humanized PD-L1/CTLA-4 bispecific antibody.

FDA Informs on Lack of Efficacy and Safety of Atezolizumab/Paclitaxel in mTNBC

The combination of atezolizumab (Tecentriq) and paclitaxel was not effective for the treatment of patients with treatment-naïve inoperable locally advanced or metastatic triple-negative breast cancer, according to an FDA alert released on September 8, 2020, to healthcare professionals and clinical trial investigators in oncology.

Plinabulin Granted FDA Breakthrough Therapy Designation for Chemotherapy-Induced Neutropenia

On September 8, 2020, a Breakthrough Therapy designation was granted to plinabulin as treatment of chemotherapy-induced neutropenia from both the FDA in the United States and China’s Center for Drug Evaluation of the National Medical Products Administration

FDA Approval Sought for Subcutaneous Daratumumab Regimen for Light Chain Amyloidosis

A supplemental Biologics License Application was submitted to the FDA on September 10, 2020, seeking approval of a subcutaneous formulation of daratumumab (Darzalex Faspro) with hyaluronidase-fihj as treatment of patients with light chain.

FDA Grants Breakthrough Therapy Designation to Toripalimab for Nasopharyngeal Carcinoma

The FDA granted a Breakthrough Therapy designation on September 10, 2020, to toripalimab for the treatment of patients with nasopharyngeal carcinoma, marking the first anti-PD-1 antibody from China to receive this designation

FDA Grants Fast Track Designation to Novel Androgen Receptor in mCRPC

On September 14, 2020, the FDA granted a Fast Track to EPI-7386, a selective androgen receptor inhibitor, which is a potential treatment for adult patients with metastatic castration-resistant prostate cancer that is resistant to standard-of-care therapy.

FDA Grants Breakthrough Therapy Designation to Margolimab in Newly Diagnosed MDS

The FDA granted a Breakthrough Therapy designation to magrolimab as treatment of patients with newly diagnosed myelodysplastic syndrome on September 15, 2020.

FDA Grants Priority Review to Ide-Cel for Relapsed/Refractory Multiple Myeloma

On September 22, 2020, the FDA granted a Priority Review designation to idecabtagene vicleucel (ide-cel; BB2121) as treatment of adult patients with multiple myeloma who have received at least 3 prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 antibody.

FDA Receives BLA for Lonca in Relapsed/Refractory Diffuse Large B-Cell Lymphoma

A Biologics License Application was submitted to the FDA on September 21, 2020, for loncastuximab tesirine (Lonca), an antibody-drug conjugate, as treatment of patients with relapsed or refractory diffuse large B-cell lymphoma.

FDA Grants Priority Review to Crizotinib in Relapsed/Refractory ALK-Positive ALCL

On September 23, 2020, the FDA accepted a supplemental New Drug Application for crizotinib (Xalkori) and granted it a Priority Review as treatment of pediatric patients with relapsed or refractory systemic anaplastic large cell lymphoma that is positive for an ALK alteration.

FDA Grants Fast Track Designation to DKN-01 in Gastric/GEJ Adenocarcinoma

The FDA granted a Fast Track designation to DKN-01 on September 24, 2020, for the treatment of patients with gastric and gastroesophageal junction adenocarcinoma whose tumors express the protein DKK1, following disease progression on or after prior treatment with fluoropyrimidine- and platinum-containing chemotherapy and, if appropriate, HER2/neu-targeted therapy.

FDA Grants Fast Track Designation to Eganelisib Combination in TNBC

On September 29, 2020, the FDA granted a Fast Track designation to eganelisib (IPI-549) in combination with an immune checkpoint inhibitor and chemotherapy for the first-line treatment of patients with inoperable locally advanced or metastatic triple-negative breast cancer.