FDA

Industry leaders share their perspectives on the guidance and how it will affect sponsors moving forward.

In an interview with ACT editor Andy Studna at DIA 2024, Beakes-Read, head, global regulatory policy and intelligence, Johnson & Johnson Innovative Medicine highlights the integration of technological advancements into drug development and benefits patients are seeing from FDA's Accelerated Approval Program.

Epkinly (epcoritamab-bysp), subcutaneously administered, T-cell engaging, IgG1-bispecific antibody, was previously granted Breakthrough Therapy Designation for the treatment of patients with relapsed or refractory follicular lymphoma.

Trial data show Dupixent rapidly and significantly improved lung function compared to placebo in adults with uncontrolled chronic obstructive pulmonary disease with type 2 inflammation.

Ocifisertib has shown significant single-agent activity in both solid and liquid tumors.

The Phase I/II MajesTEC-1 trial (NCT03145181; NCT04557098) evaluated Tecvayli (teclistamab-cqyv) at a reduced dose of 1.5 mg/kg administered every two weeks in patients with relapsed/refractory multiple myeloma.

Data from the pivotal, global, randomized, double-blind, placebo-controlled, Phase III INDIGO trial show statistically significant and clinically meaningful progression-free survival for vorasidenib treating IDH-mutant gliomas.

Trial findings show Krazati (adagrasib) plus cetuximab was well tolerated with promising clinical activity among pretreated patients with locally advanced or metastatic colorectal cancer harboring a KRASG12C mutation

Datopotamab deruxtecan, the first TROP2-directed DXd antibody drug conjugate, is being evaluated for locally advanced or metastatic nonsquamous non-small cell lung cancer.

Results from the randomized, multicenter, open-label, Phase III FLAURA 2 trial show Tagrisso plus chemotherapy delayed disease progression by nearly nine additional months in adults with locally advanced or metastatic epidermal growth factor receptor-mutated non-small cell lung cancer.

Amtagvi (lifileucel) is a personalized, one-time therapeutic option for adult patients with unresectable or metastatic melanoma who received prior treatment with a PD-1 antibody, and in patients who are BRAF V600-positive, a BRAF inhibitor with or without a MEK inhibitor.

In clinical trials, Xolair was found to significantly increase the amount of peanuts, milk, egg, and cashew that it takes to trigger an allergic reaction in participants with food allergies.

Single-arm, open-label, multicenter, non-randomized, multicohort Phase II VISION trial (NCT02864992) of Tepmetko (tepotinib) for metastatic non–small cell lung cancer shows favorable objective response rate.

There were no safety or efficacy issues identified in the FDA's complete response letter for Lymphir, with no additional trials required.

In clinical trials, patients administered Aurlumyn monotherapy experienced a significantly reduced risk of the need for amputation associated with severe frostbite.

Trials of Augtyro are ongoing for this tumor-agnostic indication, which could provide a durable treatment option for patients with NTRK-positive disease.

The Onivyde combination was evaluated in the open-label, multicenter, randomized Phase III NAPOLI 3 trial, showing significantly improved overall survival in patients with metastatic pancreatic adenocarcinoma.

Phase II trial data show positive survival results for BXCL701 combined with Keytruda (pembrolizumab) to treat patients with metastatic small cell neuroendocrine prostate cancer.

Phase IIb B-Clear trial shows bepirovirsen reduced HBsAg levels and HBV DNA following 24 weeks of treatment in patients with chronic hepatitis B.

Two multicenter, randomized, double-blind, placebo-controlled clinical trials showed that 53.1% of patients administered Eohilia achieved histologic remission compared with 1% of patients administered placebo.

Gilead announced it will discontinue the clinical development of magrolimab for hematologic cancers after the drug in combination with azacitidine plus Venclexta showed an increased risk of death related to infections and respiratory failure in patients with acute myeloid leukemia.

Data from the Phase I/II VERITAC trial (NCT04072952) showed promising findings for vepdegestrant in patients with ER-positive/HER2-negative locally advanced or metastatic breast cancer.

Phase III trial shows efficacy of Opdivo (nivolumab) plus chemotherapy followed by surgery and adjuvant Opdivo in the perioperative treatment of resectable stage IIA to IIIB non-small cell lung cancer.

Blenrep with bortezomib plus dexamethasone produced a significant improvement to median progression-free survival in the treatment of relapsed or refractory multiple myeloma compared with standard of care combination therapy.

The ongoing Phase I/II NCT05438329 trial is evaluating BNT325/DB-1305 in patients with platinum-resistant ovarian epithelial cancer, fallopian tube cancer, or primary peritoneal cancer previously administered one to three systemic treatment regimens.

The supplemental Biologics License Application for Arexvy was based on positive data from the Phase III NCT05590403 trial, which analyzed the immune response and safety of Arexvy in individuals 50-59 years of age with an elevated risk of respiratory syncytial virus-related lower respiratory tract disease.

Data from the Phase II Nivolumab and Ipilimumab Plus/minus UV1 vaccination (NIPU) clinical trial show UV1 off-the-shelf cancer vaccine plus ipilimumab (Yervoy) and nivolumab (Opdivo) improved survival in patients with unresectable malignant pleural mesothelioma.

Efineptakin alfa, the only clinical stage long-acting human interleukin-7, is currently being evaluated in Phase I/II clinical trials in combination with pembrolizumab (Keytruda) for patients with advanced solid tumors, including pancreatic cancer.

Biosyngen’s BST02 is a cell-based immunotherapy that began clinical trials in October 2023 for all types of liver cancer.

Afami-cel was developed to target the MAGE-A4 protein in synovial sarcoma, which comprises 5% to 10% of soft tissue sarcomas.