A Look Back at the FDA News from January 2022

The FDA kicked off 2022 with one approval for the treatment of select patients with uveal melanoma. An application agent the FDA planned to make a decision was voluntarily withdrawn, while another application for the same drug was submitted as a potential treatment of non­­­–small cell lung cancer.

In terms of clinical trial action, the partial clinical hold on a study of KO-539 was lifted. Another study exploring treatment with magrolimab in combination with azacitidine (Vidaza) was placed on a partial clinical hold.

In January, the FDA also granted several breakthrough therapy designations, fast track designations, and orphan drug designation other hematologic malignancies and solid tumors.

FDA Grants Rare Pediatric Disease Designation to Novel Treatment for Rhabdomyosarcoma

The FDA granted rare pediatric disease designation to IMX-110 on January 3, 2022 for the treatment of rhabdomyosarcoma, a life-threatening caner found in children. This designation makes IMX-110 eligible for a fast track designation and a priority review designation in the future.

FDA Grants BTD to Novel EGFR Inhibitor for Locally Advanced or Metastatic EGFR Exon 20+ NSCLC

On January 4, 2022, the FDA granted breakthrough therapy designation to CLN-081 for the treatment of patients with locally advanced or metastatic non-small cell lung cancer harboring epidermal growth factor exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.

FDA Grants Breakthrough Therapy Designation to Telisotuzumab Vedotin for Advanced EGFR Wild Type nsqNSCLC

The FDA granted a breakthrough therapy designation to telisotuzumab vedotin on Janaury 5, 2022 for the treatment of patients with advanced/metastatic EGFR wild type, nonsquamous non-small cell lung cancer with high levels of c-Met overexpression whose disease has progressed on or after platinum-based therapy.

FDA Grants Breakthrough Therapy Designation to Device to Treat AML-Induced Depression

On January 10, 2022, BNT200 was granted an FDA breakthrough device designation for the treatment of AML-induced depression and anxiety present during the high-intensity induction chemotherapy phase.

FDA Grants Fast Track Designation to Novel AR Inhibitor for AR+/ER+/HER- mBC

The FDA granted fast track designation to enobosarm on January 10, 2022 for the treatment of androgen receptor-positive, estrogen receptor-positive, human epidermal growth factor receptor 2-negative metastatic breast cancer patients who have shown previous disease progression on a nonsteroidal AI, fulvestrant, and CDK 4/6 inhibitor therapy, and who have AR% nuclei staining ≥ 40% in breast cancer tissue.

FDA Grants Fast Track Designation to C-CAR039 for DLBCL

On January 12, 2022, the FDA granted both a regenerative medicine advanced therapy and fast track designation to C-CAR039 for relapsed or refractory diffuse large B-cell lymphoma.

FDA Grants Fast Track Designation to CYNK-101 for HER2+ Gastric/GEJ Cancers

A fast track designation was granted by the FDA to CYNK-101 on January 18th in combination with standard frontline chemotherapy, trastuzumab (Herceptin), and pembrolizumab (Keytruda) for patients with advanced HER2-positive gastric or gastroesophageal junction adenocarcinoma.

FDA Grants Fast Track Designation to Gedatolisib for HR+/HER2- mBC

On January 19, 2022The FDA has granted fast track designation to gedatolisib, for the treatment of patients with hormone receptor-positive, HER2-negative metastatic breast cancer after progression on CDK4/6 therapy.

FDA to Review sBLA for Frontline Cemiplimab Plus Chemotherapy in Advanced NSCLC

The FDA is reviewing a supplemental biologics license application for cemiplimab-rwlc (Libtayo) in combination with chemotherapy as a first-line treatment option for patients with advanced non-small cell lung cancer on January 20, 2022.

FDA Lifts Partial Clinical Hold from KOMET-001 in R/R AML

On January 20, 2022, a partial clinical hold was lifted from the phase 1b KOMET-001 clinical trial of KO-539 in patients with relapsed or refractory acute myeloid leukemia.

FDA Grants Orphan Drug Designation to MT-601 for Pancreatic Cancer Treatment

The FDA granted orphan drug designation to MT-601 for the treatment of patients with pancreatic cancer on January 21, 2022.

FDA Grants Orphan Drug Designation to AO-176 in R/R Myeloma

The anti-CD47 antibody, AO-176, was granted an FDA orphan drug designation on January 21, 2022 for relapsed or refractory myeloma treatment and is being assessed in a phase 1/2 study.

FDA Grants Orphan Drug Designation to Eltanexor for Myelodysplastic Syndrome

On January 24, a novel oral, selective inhibitor of nuclear export compound, eltanexor, was granted orphan drug designation by the FDA for the treatment of patients with myelodysplastic syndrome.

FDA Halts Studies of Magrolimab Plus Azacitidine Amid Safety Discrepancy

On January 25, 2022, the FDA placed a partial clinical hold on studies evaluating the combination of magrolimab plus azacytidine (Vidaza) as a result of an imbalance in the investigator-reported serious adverse reaction between treatment arms.

FDA Approves Tebentafusp-tebn in Adult HLA-A*02:01-Positive Unresectable/Metastatic Uveal Melanoma

Tebentafusp-tebn (Kimmtrak)was granted FDA approval on January 26, 2022 to treat HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma based on positive results from a phase 3 study.

FDA Grants Fast-Track Designation to CX-5461 in Treatment of Certain Patients with Breast or Ovarian Cancer

The FDA granted a fast-track designation to the first-in-class G-quadruplex stabilizer CX-5461 on January 26, 2022, for the treatment of patients with BRCA1/2, PALB2, or other homologous recombination deficiency mutations in their breast or ovarian cancer.

FDA Grants Breakthrough Therapy Designation to DZD9008 for EGFR Exon20Ins-Positive NSCLC

On January 27, 2022, the FDA has granted breakthrough therapy designation to DZD9008 for the treatment of patients with locally advanced or metastatic non–small cell lung cancer with EGFR exon20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.

Cemiplimab FDA Approval Application for Recurrent or Metastatic Cervical Cancer Withdrawn

Ahead of its Prescription Drug User Fee Act target action date, the supplemental biologics license application for cemiplimab (Libtayo) as treatment of metastatic cervical cancer was voluntarilywithdrawn by the developer on January 28, 2022.