Acetylcysteine

FDA Approves Simplified Dosing Regimen for Acetadote to Enhance Acetaminophen Overdose Treatment

a month ago

• Cumberland Pharmaceuticals announced FDA approval of a simplified dosing regimen for Acetadote (N-acetylcysteine), combining the first two bags of the standard regimen into a single, slower infusion. • The streamlined approach has been shown to reduce medication errors and non-allergic anaphylactoid reactions without compromising effectiveness in treating acetaminophen overdose. • Acetaminophen poisoning is the leading cause of acute liver failure in the United States, with thousands experiencing accidental or intentional overdoses annually.

Cumberland Pharmaceuticals Reports Breakthrough in DMD Treatment and 11.6% Q4 Revenue Growth

3 months ago

• Cumberland Pharmaceuticals achieved positive Phase II results for ifetroban in treating cardiac complications of Duchenne muscular dystrophy, marking the first successful study of its kind. • The company's Q4 2024 revenue reached $10.4 million, representing an 11.6% year-over-year increase, while securing FDA Orphan Drug and Rare Pediatric Disease designations for ifetroban. • Cumberland's antibiotic Vibativ received approval in China, expanding its international presence, while the FDA approved a simplified dosing regimen for Acetadote.

Qureight's AI Imaging Platform to Support Avalyn's Inhaled Pirfenidone Trial in Pulmonary Fibrosis

4 months ago

• Qureight and Avalyn Pharma have formed a strategic partnership to integrate deep-learning imaging technology in the Phase 2b MIST trial evaluating inhaled pirfenidone (AP01) for progressive pulmonary fibrosis. • The collaboration marks the first-ever use of deep-learning imaging technologies in a progressive pulmonary fibrosis clinical study, aiming to enhance treatment response measurement. • Avalyn's inhaled pirfenidone formulation targets improved efficacy and reduced side effects compared to current oral antifibrotic therapies by delivering medication directly to affected lung tissue.

Revive Therapeutics Focuses R&D on Bucillamine for Infectious Diseases and Nerve Agent Exposure

4 months ago

• Revive Therapeutics is prioritizing the development of Bucillamine for medical countermeasures against nerve agent exposure, leveraging its antioxidant properties. • A research partnership with Defence R&D Canada is evaluating Bucillamine's potential to mitigate nerve agent-induced brain injury by enhancing GABA receptor function. • Revive is also exploring Bucillamine as a treatment for Long COVID, with plans to submit a Phase 2 study protocol to the FDA by the end of Q1 2025. • The FDA has provided feedback on study design for Long COVID, recommending a new IND application referencing data from a previous Phase 3 COVID-19 trial.

Aer Therapeutics Doses First Patient in Phase 2 Trial of Fexlamose for COPD

4 months ago

• Aer Therapeutics has initiated a Phase 2a clinical trial of fexlamose for chronic obstructive pulmonary disease (COPD) in mucus plug-high patients. • The study, AER-01-002, is a randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of fexlamose over 28 days. • The trial employs a precision imaging strategy using CT scans to identify and enroll 100 COPD patients with significant mucus plugs. • Top-line data from the study, conducted across sites in Australia, New Zealand, and potentially the UK, are expected in January 2026.

FDA Approves Simplified IV N-Acetylcysteine Regimen for Acetaminophen Overdose

5 months ago

• The FDA has approved a supplemental new drug application (sNDA) for intravenous N-acetylcysteine (NAC) to prevent or lessen liver injury from acetaminophen overdose. • The new NAC dosing regimen simplifies administration by combining the first two bags of the standard regimen into a single, slower infusion. • Studies show the simplified regimen reduces medication errors and potentially serious non-allergic anaphylactoid reactions without compromising efficacy. • Experts believe this approval marks a significant advancement in treating acetaminophen overdose, improving patient outcomes and reducing adverse events.

FDA Approves Simplified Dosing Regimen for Acetadote in Acetaminophen Overdose

5 months ago

• The FDA has approved a supplemental New Drug Application (sNDA) for Acetadote, used to prevent liver damage following acetaminophen overdose. • The new dosing regimen simplifies Acetadote administration by combining the first two doses into a single, slower infusion, reducing medication errors. • This streamlined approach, already implemented internationally, improves efficiency and patient outcomes without compromising effectiveness. • Acetaminophen overdose is a leading cause of acute liver failure in the U.S., making this approval a significant advancement.

Ifetroban Shows Promise in Improving Heart Function for DMD Patients in Phase 2 Trial

6 months ago

• Ifetroban, an oral thromboxane receptor antagonist, significantly improved left ventricular ejection fraction (LVEF) in Duchenne muscular dystrophy (DMD) patients. • The FIGHT DMD trial demonstrated a 3.3% improvement in LVEF with high-dose ifetroban, contrasting with a decline in the placebo group. • Compared to natural history controls, high-dose ifetroban showed a 5.4% overall LVEF improvement, suggesting a potential disease-modifying effect. • Ifetroban has received Orphan Drug and Rare Pediatric Disease designations, potentially becoming the first targeted therapy for DMD-related heart disease.

Molecular Profiling Reveals Drug Vulnerabilities in Pancreatic Metastases of Renal Cell Carcinoma

7 months ago

• Researchers have identified distinct molecular profiles in pancreatic metastases of clear cell renal cell carcinoma (PM-ccRCC). • Patient-derived cell cultures (PDCs) from PM-ccRCC exhibit shared drug vulnerabilities, offering potential therapeutic targets. • Exome and single-cell RNA sequencing revealed unique genetic and transcriptional features of PM-ccRCC compared to primary ccRCC. • Drug sensitivity testing identified potential therapeutic options for PM-ccRCC, highlighting personalized treatment strategies.

Insights from Failed IPF Trials Highlight Need for Adaptive Designs and Patient-Focused Outcomes

8 months ago

• Analysis of recent idiopathic pulmonary fibrosis (IPF) trial failures emphasizes the importance of learning from setbacks to refine future clinical trial designs. • Small sample sizes and short study durations in phase 2 trials can lead to overestimations of efficacy that do not translate to phase 3 studies. • Adaptive trial designs, rigorous statistical methods, and a focus on patient-relevant outcomes are crucial for improving IPF trial success. • Concurrent therapies in phase 3 trials that were not present in phase 2 can confound results.

Exploring Non-Opioid Options for Chronic Pain Management

11 years ago

• A review of studies highlights several non-opioid medications with potential benefits for chronic low back pain (CLBP), myofascial pain syndrome (MPS), and fibromyalgia (FM). • For CLBP, NSAIDs, antidepressants (duloxetine), and muscle relaxants (carisoprodol, cyclobenzaprine, diazepam) have demonstrated efficacy compared to placebo in some studies. • Pregabalin and duloxetine have shown superior pain relief compared to placebo for fibromyalgia, while lidocaine patches and BoNT-A injections may benefit myofascial pain syndrome. • Several studies suggest that combination therapies, such as nortriptyline/gabapentin and morphine/gabapentin, may offer enhanced pain relief and reduced side effects compared to monotherapy.

Drug Development Updates