A Look Back at FDA News from January 2023


Here is a roundup of all the notable FDA news in the oncology space from the month of January.

January was a busy month for the FDA and the field of oncology as the agency approved 5 therapies, including tucatinib (Tukysa), zanubrutinib (Brukinsa), pembrolizumab (Keytruda) in the later stages of non–small cell lung cancer (NSCLC), pirtobrutinib (Jaypirca), and elacestrant (Orserdu).

The agency also granted 3 orphan drug designations for novel therapies impacting patients with melanoma, skin cancer, and glioblastoma (GBM). Moreover, the FDA is continuing its review of new ways to treat patients with cancer with multiple priority reviews granted after the submission HBM1020 of biologics license applications (BLAs) for cosibelimab (formerly CK-301) and glofitamab OverC™ Multi-Cancer Detection Blood Test (RG6026).

Additionally, the FDA granted 3 fast track designations and 2 breakthrough designations to therapies in ongoing studies. The FDA also continued to approve companion diagnostics that are a part of identifying patients to their respective treatments with a breakthrough designation to the OverC™ Multi-Cancer Detection Blood Test (MCDBT).

Two clearances of studies for the treatment of advanced solid tumors and aggressive lymphomas were granted for HBM1020 and for the chimeric antigen receptor (CAR) T-cell therapy, IMPT-314, respectively.

Here’s a look back at the notable FDA updates from January 2023.

FDA Grants Breakthrough Designation for OverC™ MCDBT to Detect Early Cancers

On January 3rd, 2023, the agency granted a breakthrough device designation to the OverC™ MCDBT for the early detection of esophageal, liver, lung, ovarian, and pancreatic cancers in adult patients aged to 50-75 years with average risk. Findings from the THUNDER (NCT04820868) case-control study showed that the use of the MCDBT had a 98.9% rate of specificity and 69.1% rate of sensitivity.

FDA Approval Sought for Cosibelimab as cSCC Treatment 

Due to the findings of an ongoing phase 1 trial (NCT03212404), Checkpoint Therapeutics submitted a BLA on January 4, 2023, for cosibelimab to treat patients with metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that are not candidates for curative surgery or radiation. The approval is sought for a fixed dose of cosibelimab given at 800 mg every 2 weeks or 1200 mg every 3 weeks.

FDA Approves FoundationOne Companion Diagnostic to Entrectinib in NSCLC

In order to identify if patients with NSCLC are positive for the ROS1 mutation, and therefore eligible for treatment with entrectinib (Rozylytrek), the FDA approved the use of the FoundationOne Liquid CDx tool on January 4, 2023. The comprehensive genomic profiling test allows for a wider subset of patients to be tested and potentially matched to their right treatment, as ROS1 gene fusions are seen in 1%-2% of NSCLC.

BT8009 Receives Fast Track Designation for Advanced Urothelial Cancer

On January 4, 2023, the FDA also granted fast track designation to BT8009, a Bicycle Toxin Conjugate (BTC), as monotherapy for adults with previously treated locally advanced or metastatic urothelial carcinoma.

FDA Grants Orphan Drug Designation for JBI-802 in SCLC and AML

An orphan drug designation was granted to the dual epigenetic modulator JBI-802 to treat patients with small cell lung cancer and acute myeloid leukemia on January 5, 2023. The agent works to achieve optimal inhibition of the transcriptional regulator CoREST, which regulates the development of cellular lineages responsible for neuroendocrine tumors.

FDA Accepts BLA and Grants Priority Review for Glofitamab in R/R LBCL

On January 6, 2023, the FDA accepted another BLA and granted a priority review for the investigational CD20xCD3 T-cell engaging bispecific antibody glofitamab (RG6026) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after 2 or more lines of systemic therapy. The review comes from the findings of the pivotal phase 1/2 NP30179 study (NCT03075696) which showed the durability of glofitamab. Enrolled patients achieved a 40% complete response.

FDA Grants Orphan Drug Designation to Azeliragon for Patients with Glioblastoma

The FDA granted another orphan drug designation on January 9, 2023, to azeliragon (formerly TTP488) for patients with a GBM, a once-a-day pill that is a small molecule antagonist of the receptor for advanced glycation endproducts (RAGE), and inhibits RAGE interactions with certain ligands in the GBM environment. By preventing this interaction, azeliragon may inhibit GBM and overcome resistance to effective treatment.

FDA Grants Fast Track Designation to Novel TKK1 Therapy for ER+/HER2- Advanced Breast Cancer

On January 10th, 2023, the FDA granted a fast-track designation for the HPK1 inhibitor CFI-402257 for the treatment of adult patients with estrogen receptor (ER)-positive/HER2-negative advanced breast cancer after disease progression on prior CDK4/6 inhibitors and endocrine therapy. CFI-402257 is being looked at as both a monotherapy and in combination with fulvestrant, and is now being evaluated as a phase 1 dose-confirming study (NCT05251714).

FDA Grants Breakthrough Device Designation to OncobiotaLUNG Assay for Lung Cancer Detection

The OncobiotalLUNG assay from device maker Micronoma was granted a breakthrough device designation on January 10, 2023, for the early detection of lung cancer. The microbiome-driven liquid biopsy platform categorizes lung nodule samples into those that are considered low or high-risk for disease based off the blood draw of a patient. The hope with this device is to cast a wider net when detecting lung cancer and avoid invasive biopsy. Further analysis is now under way.

FDA Clears IND Application for HBM1020 for Advanced Solid Tumors

On January 12, 2023, the FDA cleared an investigational new drug (IND) application for clinical trials of HBM1020 as a treatment option for patients with advanced solid tumors. The first-in-class fully human monoclonal antibody targets B7H7, which is a newly discovered novel immunomodulatory molecule that belongs to the B7 family, and works to regulate the T-cell response.

FDA Approves Tucatinib Plus Trastuzumab for RAS Wild-Type, HER2+ mCRC

The agency approved tucatinib (Tukysa) in combination with trastuzumab (Herceptin) on January 19, 2023, for adult patients with RAS wild-type, HER2-positive unresectable or metastatic colorectal cancer that progressed following treatment with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. The approval comes from the findings from the phase 2 MOUNTAINEER study (NCT03043313) that showed the combination to elicit a confirmed objective response rate of 38.1% (95% CI, 27.7%-49.3%) and a median duration of response of 12.4 months (95% CI, 8.5-20.5).

FDA Approves Zanubrutinib in Adult Patients with CLL/SLL

Also on January 19, 2023, the FDA approved the Bruton's tyrosine kinase (BTK) inhibitor zanubrutinib (Brukinsa) for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic leukemia (SLL). This was based on the data from the phase 2 phase 3 randomized studies that looked at the comparison of zanubrutinib vs ibrutinib (Imbruvica) and zanubrutinib vs bendamustine and rituximab for treatment-naïve patients with CLL in the ALPINE study (NCT03734016) and SEQUOIA study (NCT03336333), respectively.

FDA Grants Orphan Drug Designation to LNS8801 for Metastatic Cutaneous Melanoma

An orphan drug designation was granted to LNS8801 on January 19, 2023, for the treatment of patients with metastatic cutaneous melanoma. Preclinical models have shown the agent to elicit potent antitumor activity across multiple tumor types, rapidly shrink tumors, and induce immune memory.

FDA Grants Fast Track Designation to DB-1303 for Advanced HER2-Expressing Endometrial Cancer

On January 20, 2023, the FDA granted fast track designation to DB-1303 for the treatment of patients with advanced, recurrent, or metastatic endometrial carcinoma with HER2 overexpression, who have progressed on or after receiving standard systemic treatment. This was granted in part due to the significant unmet need for treatments that address failure on previous treatment.

FDA Grants Priority Review to Avapritinib for Indolent Systemic Mastocytosis

On January 23, 2023, the FDA granted a priority review to a supplemental new drug application for avapritinib (Ayvakit), a potential treatment option for adult patients with indolent systemic mastocytosis, based on findings from the phase 2 PIONEER study (NCT03731260).

FDA Clears Study of Novel CAR T-Cell Therapy for Aggressive B-Cell Lymphoma

The FDA cleared another study on January 24, 2023 for the bispecific autologous CAR T-cell therapy IMPT-314 for the treatment of patients with aggressive B-cell lymphoma, including diffuse large B-cell lymphoma. The therapy targets the B-cell antigens CD19 and CD20 and now, a phase 1/2 study beginning early 2023.

FDA Approves Adjuvant Pembrolizumab for Stage IB-IIIA NSCLC

On January 26, 2023, the FDA approved the use of adjuvant pembrolizumab (Keytruda) after the resection and platinum-based chemotherapy for stage IB (T2a ≥4 cm), II, or IIIA NSCLC. The approval comes off the heels of the phase 3 PEARLS/KEYNOTE-091 trial (NCT02504372) that showed that the treatment led to improved disease-free survival compared with placebo.

FDA Green Lights Fast Track Designation to Tamibarotene for Higher-Risk MDS

Also on January 26, 2023, the FDA granted a fast track designation to the first-in-class selective retinoic acid receptor alpha agonist tamibarotene (Amnolake) for the treatment of patients with higher-risk myelodysplastic syndrome (HR-MDS). The agent is currently under investigation in combination with azacitidine (Vidaza) for patients with HR-MDS who have RARA gene overexpression, and early results have been promising to the agency.

FDA Approves Pirtobrutinib for Patients With MCL

On January 27, 2023, the FDA granted an approval to pirtobrutinib (Jaypirca) for the treatment of adult patients with relapsed/refractory mantle cell lymphoma (MCL) who previously received at least 2 lines of systemic therapy, including a BTK inhibitor. With this approval, pirtobrutinib is the first BTK inhibitor of any kind to be approved for patients with MCL treated with a prior covalent BTK inhibitor.

FDA Approves Elacestrant for ER+/HER2- Advanced Breast Cancer

The agency granted another approval on January 27, 2023, for elacestrant (Orserdu), which is the first oral selective estrogen receptor degrader to show an improved efficacy over the standard of care for patients with ER-positive/HER2-negative advanced or metastatic breast cancer.

FDA Okays Guardant360 Test as CDx for Elacestrant in ESR1-mutated Breast Cancer

On January 30, 2023, the FDA approved the Guardant360 test as a companion diagnostic for patients that could be potentially matched to elacestrant (Orserdu) for the treatment of their advanced or metastatic breast cancer harboring ESR1 mutations. Guardant360 CDx is also a blood test that is a comprehensive genomic profiling that showed effectiveness for this patient population.

FDA Grants Breakthrough Therapy Designation to Pimicotinib for TGCT

On January 31, 2023, the FDA granted breakthrough therapy designation to the CSF-1R inhibitor, pimicotinib (ABSK02), for the treatment of patients with tenosynovial giant cell tumor who are not amenable to surgery.

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