March 2023 Brief

Azeliragon has been granted an orphan drug designation by the FDA for patients with glioblastoma after previous trials demonstrated the agent to be well-tolerated in Alzheimer’s disease.

With statistically significant and clinically meaningful event-free survival improvement in patients with stage II-IIIB Non–small cell lung cancer, could perioperative pembrolizumab in combination with chemotherapy become a new treatment option?

Data from the second interim analysis of the phase 3 MAGNITUDE study showed that the addition of niraparib to abiraterone acetate and prednisone prolonged survival for patients with metastatic castration-resistant prostate cancer with BRCA mutations.

The FDA has planned a regular review of the biologic license application for cosibelimab for the treatment for metastatic or locally advanced cutaneous squamous cell carcinoma.

With a broadened FDA label for abemaciclib, patients high-risk breast cancer can now be categorized based only on nodal status, tumor size, and tumor grade.

The VENTANA PD-L1 Assay can now be used to detect PD-L1 expression in patients with non–small cell lung cancer to determine eligibility for treatment with cemiplimab.

The injectable formulation of nelarabine has been granted FDA approval to treatment 2 hematologic malignancies.

Findings from a phase 3 clinical trial, including data from high-risk children given remestemcel-L, led the FDA to accept the biologics license application of the therapy.

The FDA has granted the cell therapy temferon an orphan drug designation to treat patients with brain cancer.

Following positive phase 1 results in patients with previously treated advanced urothelial carcinoma, the FDA granted Fast Track Designation for the novel AHR inhibitor IK-175.

In a vote of 11 to 2, the FDA's Oncologic Drugs Advisory Committee voted for the benefit/risk profile of polatuzumab vedotin in combination with R-CHP, based on findings from the confirmatory POLARIX trial.

Soligenix has requested a Type A meeting with the FDA to get clarity around the item needed to complete the new drug application of SGX301 for patients with early-stage cutaneous T-cell lymphoma.

A comprehensive analytical data package and pharmacokinetic, pharmacodynamic, and immunogenicity study has led the FDA to approve the pegfilgrastim biosimilar pegfilgrastim-cbqv for patients with cancer.

As a result of the FDA’s clinical hold on the phase 1 trial, no patients with HER2-positive recurrent or metastatic solid tumors will be enrolled or dosed with XMT-2056.

Ripretinib now has 2 breakthrough therapy designations from the FDA and has been added to the NCCN guidelines for gastrointestinal stromal tumor.

The newly approved supplemental new drug application for Illuccix by the FDA will enable patients with prostate cancer to be identified and selected for radioligand therapy.

Findings from the phase 2 Study CDRB436G2201 trial have led the FDA to approve dabrafenib combined with trametinib for pediatric patients with BRAF V600E-mutated low-grade glioma who require systemic therapy.

PBP1510 has received a fast track designation from the FDA for patients with unresectable or metastatic pancreatic adenocarcinoma that has relapsed following and/or is refractory to at least 1 prior line therapy.

ISB 1442, a 2+1 biparatopic bispecific antibody that has CD38 and CD47 targeting domains, has received an orphan drug designation from the FDA and is being assessed in a phase 1/2 clinical trial for patients with multiple myeloma.

The FDA has okayed PD-1 inhibitor for the treatment of adults with advanced Merkel cell carcinoma.

The first orphan drug designation from the FDA has been received by FORE Biotherapeutics and for the FORE8394 program for patients with primary brain and central nervous system malignancies.

Tirabrutinib has received orphan drug status from the FDA and is being studied in the phase 2 PROSPECT study for patients with primary central nervous system lymphoma.

The FDA noted that additional requirements are needed prior to the approval of ruxolitinib extended-release for patients with myelofibrosis, polycythemia vera, and graft-versus-host disease, in a complete response letter.

The FDA released a draft guidance for drug sponsors of clinical trials in oncology, outlining its move toward requiring randomized controlled trials to grant accelerated approvals for most oncology therapeutics.

As of March 28, 2023, the partial clinical hold on the phase 1/2 VELA trial, which is evaluating the selective CDK2 inhibitor BLU-222 for patients with solid tumors, has been lifted. Blueprint Medicines has resumed the re-initiation of patient enrollment.

A full approval has been granted to pembrolizumab by the FDA for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high or mismatch repair deficient solid tumors.

The FDA has granted a fast-track designation for RRx-001 and accepted the investigational new drug application for a phase 2b trial of severe oral mucositis in chemotherapy- and radiation-treated patients with head & neck cancer.

A phase 1/2 trial investigating GRC 54276 for the treatment of patients with advanced solid tumors and lymphomas will begin following the acceptance of an investigational new drug application for the agent.