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The role of belantamab mafodotin for patients with relapsed and/or refractory multiple myeloma.

Belantamab mafodotin (belamaf) is a first-in-class anti-B-cell maturation antigen (BCMA) antibody-drug conjugate (ADC) that recently gained regulatory approval for the treatment of relapsed and/or refractory multiple myeloma (RRMM) patients who have received at least four prior therapies including an anti-CD38 monoclonal antibody (mAb), a proteasome inhibitor (PI), and an immunomodulatory drug (IMiD). As the first BCMA-targeted therapy to be approved in multiple myeloma along with its "off-the-shelf" outpatient administration, belamaf addresses a significant unmet need in RRMM that is refracto

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… While the ORR of 31% in the 2.5 mg/kg cohort in the DREAMM-2 study is comparable to other recent single-agent myeloma drug approvals, 25 – 27 the depth (⩾VGPR 19%) and durability of responses (median 11 months) were particularly encouraging. … Conclusion The regulatory approval of belamaf represents a significant advance as the first-in-class ADC and first BCMA-targeted drug approved for the treatment of RRMM based on data from the pivotal DREAMM-2 study. …

Recent Advances in Intravesical Chemotherapy: Insights into the FDA-Approved Gemcitabine-Releasing TAR-200 System for Bladder Cancer Management.

The FDA approval of the gemcitabine intravesical system (Inlexzo/TAR-200) marks a breakthrough for BCG-unresponsive NMIBC with CIS. This novel drug-device combination enables sustained intravesical chemotherapy, achieving high complete response rates with acceptable safety. It represents a paradigm shift in bladder-sparing therapy, though long-term outcomes and accessibility remain critical considerations.

Hitting a New Target in Multiple Myeloma.

ONCOLOGY sat down with Sagar Lonial, MD, professor and chair of Hematology and Medical Oncology at the Emory University School of Medicine and chief medical officer at Emory's Winship Cancer Institute, to discuss the FDA approval of the antibody-drug conjugate belantamab mafodotin, along with other BCMA-targeting agents for patients with multiple myeloma.

Research and development spending versus revenues after approval for CFTR modulators.

BACKGROUND: The high prices of CFTR modulators present barriers to access for people with cystic fibrosis (CF), especially those in low- and middle-income countries. Costs of research and development (R&D) are often cited as key drivers of drug pricing. In the case of CFTR modulators, circumstances allow for a meaningful comparison of pre-marketing R&D spending and post-approval product revenues. METHODS: Data on R&D expenditure and CFTR modulator revenues from Q3 2001 (the initial acquisition of CFTR modulator research) until 2024 were extracted from the originator company's Securities and E

Utilization trends of cervical artificial disc replacement after FDA approval compared with anterior cervical fusion: adoption of new technology.

STUDY DESIGN: Epidemiologic study. OBJECTIVE: To compare the utilization of anterior cervical discectomy and fusion (ACDF) versus cervical disc arthroplasty (CDA) in terms of patient and hospital characteristics during the 3 years after Food and Drug Administration (FDA) approval of CDA devices in 2007. SUMMARY OF BACKGROUND DATA: There was a surge in CDA adoption in the 3 years prior to FDA approval of CDA devices in 2007. However, utilization trends of CDA versus ACDF since the FDA approval are unknown. METHODS: The Nationwide Inpatient Sample database was used to identify CDA and ACDF pr

Drug adjuncts for treating alcohol dependence.

Three drugs are approved by the US Food and Drug Administration for treating alcoholism: disulfiram, naltrexone, and acamprosate. Drugs approved for other indications that are being used experimentally or "off-label" include nalmafene, topiramate, and ondansetron. As we learn more about the pathophysiologic basis of alcoholism, it is hoped that novel drugs can be developed to help people with alcohol dependence achieve abstinence, and as a result, curb alcohol-related morbidity.

The Expendables: Overlooked Medications with Anti-Cancer Properties.

PURPOSE OF REVIEW: Cancer is a leading cause of death worldwide. The process to develop new anti-cancer drugs is long and arduous. Therefore, additional strategies for drug development could involve drug repositioning, i.e., identifying new uses for already approved medications that have been deemed expendable in the oncologic treatment realm. Indeed, there are already drugs that have proven that they are extendable into oncology and are now Food and Drug Administration (FDA) approved for malignancies, though they were initially developed for other conditions. RECENT FINDINGS: These drugs inc

[Discovering L-type calcium channels inhibitors of antihypertensive drugs based on drug repositioning].

This study was amid to construct the pharmacophore model of L-type calcium channel antagonist in the application of screening Drugbank and TCMD. This paper repositions the approved drugs resulting from virtual screening and discusses the relocation-based drug discovery methods, screening antihypertensive drugs with L-type calcium channel function from TCMD. Qualitative hypotheses wre generated by HipHop separately on the basis of 12 compounds with antagonistic action on L-type calcium channel expressed in rabbit cardiac muscle. Datebase searching method was used to evaluate the generated hypot

Talk to Patients About: Meningococcal B.

Meningococcal B (MenB) vaccine is the new kid on the block for children and adolescents, having won Food and Drug Administration approval in 2014. This presents a problem for physicians: Because there is an older vaccine for the other types of meningococcal bacteria, many patients who've had that vaccine wrongly believe they're also protected against MenB.

Comparison of the United Kingdom and United States approaches to approval of new neuromuscular therapies.

Many novel therapies are now available for rare neuromuscular conditions that were previously untreatable. Hereditary transthyretin amyloidosis and spinal muscular atrophy are two examples of diseases with new medications that have transformed our field. The United States and the United Kingdom have taken disparate approaches to the approval and coverage of medications, despite both providing incentives to develop therapies targeting rare diseases. The US requires less evidence for approval when compared with medications for common diseases and does not have a mechanism to ensure or even encou

What's new in clinical pharmacology and therapeutics.

The US Food and Drug Administration (FDA) has approved several new drugs in the last few years. We have summarized a few of these that should be of interest to a primary care physician. These belong to either a new class of drugs or have a better drug profile in terms of ease of administration, prolonged duration of action, or fewer side effects. Daptomycin is a cyclic lipopeptide, active against methycillin resistant Staphylococcus aureus (MRSA). Telithromycin is a ketolide that can be used in place of macrolide antibiotics. Rifaximin is a semi-synthetic derivative of rifampin approved by the

Olumniant (Baricitinib) oral tablets: An insight into FDA-approved systemic treatment for Alopecia Areata.

Olumniant (Barcitinib) has gained swift approval by the Food and Drug Administration (FDA) as a promising medication for the treatment of adults with severe Alopecia Areata. This drug has proven to be effective for various conditions including Covid-19 and Rheumatoid Arthritis as well as being found to be less costly and minimally invasive, with no need for post-operative management. However, its use has some considerable potential implications before its prescription can be made commonplace. Therefore, more trials need to be conducted to establish a more rigid safety profile.

The impact of GLP-1 agonists on sleep disorders: spotlight on sleep apnea.

INTRODUCTION: For the first time in history, a medication - tirzepatide, has been approved by the United States Food and Drug Administration (FDA) for the treatment of obstructive sleep apnea (OSA). This approval adds to the current array of therapeutic options and raises many issues regarding the future of treatment for OSA; in particular, the role of glucagon-like peptide-1 receptor agonists (GLP-1 RAs) in the chronic management of patients with OSA. AREAS COVERED: This review aims to summarize the impact of GLP-1 RAs for the treatment of OSA in the context of the recent FDA approval of tir

Checkpoint immunotherapy by nivolumab for treatment of metastatic melanoma.

Clinical management of metastatic melanoma suffered historically from a lack of effective targeted and immunotherapies due to short-lived clinical responses. Recent advances in our understanding of tumor-immune signaling pathways, discovery of immunosuppressive checkpoints, and subsequent development of antibodies that target these checkpoints reverses the situation to some extent. Two antibodies ipilimumab and nivolumab gained Food and Drug administration approval for the treatment of metastatic melanoma and target two major immunosuppressive checkpoints cytotoxic T lymphocyte antigen and pro

Simeprevir: first global approval.

Simeprevir (Sovriad(TM)) is a new direct-acting antiviral drug and a second-generation small-molecule NS3/4A serine protease inhibitor developed by Janssen and Medivir for the oral treatment of adults with genotype 1 and/or genotype 4 chronic hepatitis C virus (HCV) infection (chronic hepatitis C). Simeprevir antiviral activity is achieved by its non-covalent binding to HCV protease, with a fast association and slow dissociation rate. The capsule formulation is approved in Japan and Canada for use in combination with pegylated interferon (peginterferon) and ribavirin for genotype 1 chronic hep

Revumenib-Induced QTc Prolongation Leading to Multiple Episodes of Torsades de Pointes and Ventricular Arrhythmias.

BACKGROUND: Revumenib is the only oral menin inhibitor approved for the treatment of relapsed/refractory acute leukemia with menin-lysine methyltransferase 2A (KMT2A) rearrangement in adults and pediatric patients aged >1 year. Revumenib was granted US Food and Drug Administration approval under the accelerated approval pathway, receiving both breakthrough therapy and orphan drug designations because of its potential to address a significant unmet need for patients with relapsed/refractory acute leukemia with a KMT2A translocation. While revumenib has no absolute contraindications, the package

The Promise of Therapeutic Psilocybin: An Evaluation of the 134 Clinical Trials, 54 Potential Indications, and 0 Marketing Approvals on ClinicalTrials.gov.

INTRODUCTION: Psilocybin, a tryptamine psychedelic, has been touted in the media both historically and recently as a potential game-changing mental health therapeutic. ClinicalTrials.gov has over one hundred and thirty psilocybin clinical trials listed covering the last twenty years. The single most important aspect of any therapeutic is to gain approval for marketing and thus enter the real-world phase of development. A typical new chemical entity progresses from inception to US Food and Drug Administration (FDA) approval in approximately 12 years and seeks approval for a single indication.

The possibility of obtaining marketing authorization of orphan pharmaceutical compounding preparations: 3,4-DAP for Lambert-Eaton Myasthenic Syndrome.

BACKGROUND: Pharmaceutical compounding preparations, produced by (hospital) pharmacies, usually do not have marketing authorization. As a consequence, some of these pharmaceutical compounding preparations can be picked-up by a pharmaceutical company to obtain marketing authorization, often leading to price increases. An example is the 3,4-diaminopyridine slow release (3,4-DAP SR) tablets for Lambert-Eaton Myasthenic Syndrome (LEMS). In 2009 marketing authorization was given for the commercial immediate release phosphate salt of the drug, including a fifty-fold price increase compared to the ph

The safety of newly approved medicines: do recent market removals mean there is a problem?

The removal of 5 pharmaceuticals from the market in a 12-month period because of unexpected adverse events raised concerns about the adequacy of the drug review process at the US Food and Drug Administration (FDA). Specifically, concerns were raised about improvements in drug review efficiency that significantly reduced FDA review times. We have reviewed the circumstances of the 5 removals to determine whether there was any relationship to the increased efficiencies in the drug review process. When the removed drugs were analyzed by date of approval, no increase in the number of drugs taken of

Sutimlimab for cold agglutinin disease: an updated perspective from approval to real-world clinical treatment.

INTRODUCTION: Sutimlimab, a classical complement pathway (CP) inhibitor, was approved in 2022 in the US, EU, and Japan for the treatment of cold agglutinin disease (CAD), a rare form of autoimmune hemolytic anemia (AIHA) characterized by CP‑mediated extravascular hemolysis and circulatory symptoms related to IgM‑mediated red blood cell (RBC) agglutination. This review reexamines the clinical trial data and compares those findings with published real‑world experience (RWE), providing clinicians with efficacy and safety data that extend beyond the clinical trial experience in this rare AIHA. AR