Articles related to DEVICE/DRUG APPROVALS

Demonstrating compelling efficacy in the EV-103 trial, the combination of enfortumab vedotin-ejfv and pembrolizumab emerges as a promising treatment for patients with locally advanced or metastatic urothelial carcinoma ineligible for cisplatin-based chemotherapy. The recent approval of enfortumab vedotin-ejfv in combination with pembrolizumab for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) who are ineligible for cisplatin-based chemotherapy was substantiated by compelling results from the multi-cohort Phase 1b/2 EV-103 trial. In the dose escalation cohort and cohort A of the trial, patients received this innovative combination therapy. In cohort K, patients were randomized to receive either the combination or enfortumab vedotin-ejfv monotherapy. The main efficacy outcome measures were the objective response rate (ORR) and duration of response (DOR), with the ORR reaching 68% (95% CI, 58.7-76.0) among the 121 patients who received the combination therapy. Moreover, the median DOR was reported as 22.1 months for the dose escalation and cohort A. Nevertheless, patients did report a range of adverse reactions, most commonly increased glucose, increased aspartate aminotransferase, rash, and decreased hemoglobin, among others. The approval of this therapy is dependent on the validation and depiction of clinical benefits from the ongoing Phase 3 EV-302 confirmatory trial. Given the promising ORR […]

The FDA has granted approval for Otsuka Pharmaceutical’s brexpiprazole as the first drug to treat agitation in patients with Alzheimer’s disease. Agitation is a common symptom in Alzheimer’s patients, characterized by extreme aggression, restlessness, and anxiety. Previously, non-pharmacological strategies were used initially, followed by off-label use of antipsychotics and anti-depressants in severe cases. The decision to approve brexpiprazole was based on two late-stage studies demonstrating its effectiveness in calming agitated patients with Alzheimer’s, compared to a placebo. The drug was already approved for the treatment of major depressive disorder and schizophrenia in adults. This approval addresses an unmet need in the management of agitation in Alzheimer’s patients and provides healthcare professionals with a new pharmacological option. Brexpiprazole has shown significant improvement in calming agitated patients, offering a potential solution to help alleviate symptoms associated with the disease. The drug’s approval was supported by sufficient data and endorsement from an advisory panel, reinforcing its benefits outweigh the associated risks.

During its April 28 meeting, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 11-1 on whether access to the combination should be limited to patients whose tumors carry BRCA mutations. The majority of members shared the FDA reviewers’ concerns regarding clinical trial results and uncertainty about whether people without the BRCA mutation would benefit from the regimen. The FDA argued that the results of the phase 3 PROpel clinical trial could not be applied to patients who had not had their BRCA or HRR mutation status determined. As a result, the FDA contended that the PROpel intention-to-treat (ITT) group was heterogeneous, complicating the interpretation and application of the trial’s results to unselected patients.

The approval was based on results from a 24-week open-label, two-part phase 3 trial that assessed Trikafta’s effectiveness and safety in 75 CF patients having at least one F508del mutation or a mutation known to be responsive to Trikafta. According to the findings, patients’ sweat chloride concentration and lung clearance index decreased in a clinically significant way. Trikafta therapy was typically well tolerated and commensurate with results seen in individuals who were older.

The FDA has authorized the use of the mRNA bivalent COVID-19 vaccine (Moderna and Pfizer-BioNTech) for all doses administered to individuals 6 months of age and older. The monovalent vaccines are no longer authorized for use in the US. This simplifies the vaccination schedule and encourages future vaccination. For child vaccination, unvaccinated children may receive a 2-dose series of the Moderna bivalent vaccine (6 months through 5 years) or a 3-dose series of the Pfizer-BioNTech bivalent vaccine (6 months through 4 years). Children who are 5 years old may receive 2 doses of Moderna bivalent vaccine or a single dose of Pfizer-BioNTech bivalent vaccine. For immunocompromised children 6 months through 4 years, eligibility depends on the vaccine previously received. For individuals 5 years of age and older with certain kinds of immunocompromise, a single additional dose of bivalent vaccine may be administered at least 2 months following the initial dose of a bivalent vaccine. Additional doses may be administered at the discretion of the health care provider, taking into consideration the individual’s clinical circumstances.

The FDA’s Oncologic Drugs Advisory Committee (ODAC) has voted to support the use of polatuzumab vedotin-piiq (Polivy) in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (Pola+R-CHP) for the first-line treatment of patients with previously untreated large B-cell lymphoma (LBCL). The committee voted 11-2 in favour of the drug following the confirmatory POLARIX trial, a double-blind, placebo-controlled, phase 3 study, which enrolled 879 patients with LBCL, including diffuse large B-cell lymphoma (DLBC), not otherwise specified (NOS). Although concerns were raised about modest benefits in progression-free survival (PFS) and similar overall survival (OS) between Pola+R-CHP and the standard treatment R-CHOP, ODAC member Neil Vasan, MD, PhD, said that the benefits outweighed the risks and Pola+R-CHP should be an option for first-time treatment of DLBCL with curative intent, as it could spare patients from more toxic and complicated salvage therapies.