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U.S. Food and Drug Administration Approval: peginterferon-alfa-2b for the adjuvant treatment of patients with melanoma.

On March 29, 2011, the U.S. Food and Drug Administration approved peginterferon alfa-2b (PEG-IFN) (Sylatron™; Schering Corporation, Kenilworth, NJ) for the adjuvant treatment of melanoma patients with microscopic or gross nodal involvement following definitive surgical resection including complete lymphadenectomy. The approval was based on a single, open-label, multicenter trial enrolling 1,256 patients. After surgical resection, patients were randomized (1:1) to either PEG-IFN or observation for 5 years. PEG-IFN, 6 μg/kg per week, was administered s.c. for eight doses, followed by 3 μg/kg per

Association Between Survival Benefits of Anticancer Drugs and Probability of US Food and Drug Administration Approval: A Systematic Review.

BACKGROUND AND OBJECTIVES: Survival benefit constitutes the primary pillar of therapeutic efficacy in oncology. However, the survival benefits observed in registrational trials broadly range from significant to marginal, even for US Food and Drug Administration (FDA)-approved drugs. This study explores the association between survival benefits and the likelihood of FDA approval and estimates the boundary effect size that distinguishes FDA-approved from non-approved drugs.MethodsWe screened 3463 phase 3 trials initiated between 1990 and 2021 on ClinicalTrials.gov. Eligibility was restricted to

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… In the new drug approval process, the United States (US) Food and Drug Administration (FDA) individually evaluates the clinical data for the investigational drug, considering the specific therapeutic context. … Furthermore, as our analysis focused on OS-powered trials, the findings may not be generalizable to drugs approved based on surrogate endpoints. …

In Vitro Screening of a 1280 FDA-Approved Drugs Library against Multidrug-Resistant and Extensively Drug-Resistant Bacteria.

Alternative strategies against multidrug-resistant (MDR) bacterial infections are suggested to clinicians, such as drug repurposing, which uses rapidly available and marketed drugs. We gathered a collection of MDR bacteria from our hospital and performed a phenotypic high-throughput screening with a 1280 FDA-approved drug library. We used two Gram positive (Enterococcus faecium P5014 and Staphylococcus aureus P1943) and six Gram negative (Acinetobacter baumannii P1887, Klebsiella pneumoniae P9495, Pseudomonas aeruginosa P6540, Burkholderia multivorans P6539, Pandoraea nosoerga P8103, and Esche

An Overview of Cancer Drugs Approved by the US Food and Drug Administration Based on the Surrogate End Point of Response Rate.

ImportanceApproximately one-third of cancer drugs are approved based on response rate (RR)-the percentage of patients whose tumors shrink beyond an arbitrary threshold-typically assessed in a single-arm study.ObjectiveTo characterize RR end points used by the US Food and Drug Administration (FDA) for cancer drug approval.Design, setting, and participantsA retrospective review of FDA-approved drug indications in oncology from 2006 to 2018.ExposuresData related to cancer type, line of therapy (first-line, second-line, or third-or-later-line treatment for advanced/metastatic disease), type of FDA…

Folotyn (pralatrexate injection) for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma: U.S. Food and Drug Administration drug approval summary.

PURPOSE: On September 24, 2009, the U.S. Food and Drug Administration granted accelerated approval for Folotyn (pralatrexate injection, Allos Therapeutics, Inc.) as a single agent for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL); it is the first drug approved for this indication. EXPERIMENTAL DESIGN: This review was based on study PDX-008, a phase II, single-arm, nonrandomized, open-label, international, multicenter trial, designed to evaluate the safety and efficacy of pralatrexate when administered concurrently with vitamin B(12) and folic acid sup

Model-Informed Drug Development Supports Full Approval of Ibuprofen Injection in Chinese Pediatric Patients With Fever or Pain.

Ibuprofen is a commonly used nonsteroidal anti-inflammatory drug, and its injectable form has specific clinical applications. However, ibuprofen injection has not been approved for use in Chinese children. This study aimed to support its approval in this population using a model-informed drug development (MIDD) approach. Ibuprofen injection (Fenliping®) received approval for Chinese pediatric indications in 2019, with a clinical trial waiver based on pediatric extrapolation using modeling and simulation. A physiologically based pharmacokinetic (PBPK) model was used to assess ethnic differences

Approval of Raxibacumab for the Treatment of Inhalation Anthrax Under the US Food and Drug Administration "Animal Rule".

On December 14, 2012, the FDA approved Raxibacumab, the first monoclonal antibody product developed under Project BioShield to achieve this milestone, and the first biologic product to be approved through the FDA animal efficacy rule (or "Animal Rule"). Raxibacumab is approved for the treatment of adult and pediatric patients with inhalational anthrax due to Bacillus anthracis in combination with appropriate antibiotic drugs and for prophylaxis of inhalational anthrax when alternative therapies are not available or not appropriate. The developmental process required for approval of Raxibacumab

Is there a path for approval of an antiobesity drug: what did the Sibutramine Cardiovascular Outcomes Trial find?

PURPOSE OF REVIEW: Obesity continues to increase in prevalence in the USA and throughout the world. It is clearly a major contributor to morbidity and mortality. Unfortunately, effective prevention strategies are few. As a contributor to cardiovascular disease, obesity is an important treatment objective. However, before approval, all drugs must meet safety and efficacy concerns of the US Food and Drug Administration. RECENT FINDINGS: Since July 2010, the Food and Drug Administration's Endocrine and Metabolic Advisory Committee has reviewed three new drug applications and one previously appro

FDA drug approval summary: pegaspargase (oncaspar) for the first-line treatment of children with acute lymphoblastic leukemia (ALL).

On July 24, 2006, the U.S. Food and Drug Administration granted approval to pegaspargase (Oncaspar; Enzon Pharmaceuticals, Inc., Bridgewater, NJ; hereafter, O) for the first-line treatment of patients with acute lymphoblastic leukemia (ALL) as a component of a multiagent chemotherapy regimen. O was previously approved in February 1994 for the treatment of patients with ALL who were hypersensitive to native forms of L-asparaginase. The trial supporting this new indication was an open label, randomized, multicenter clinical trial that enrolled 118 children (age, 1-9 years) with previously untrea

Food and Drug Administration Approval Summary: Odevixibat (Bylvay) for the Treatment of Pruritus With Progressive Familial Intrahepatic Cholestasis.

On July 20, 2021, the Food and Drug Administration approved odevixibat (Bylvay) for the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare disease that results in impaired bile secretion and transport, leading to cholestatic liver injury. Odevixibat is a reversible inhibitor of the ileal bile acid transporter. It decreases the reabsorption of bile acids from the terminal ileum (distal small intestines). Approval was based on the improvement in pruritus demonstrated in a 24-week randomized double-blind placebo-co

Physician Perspectives on the Food and Drug Administration's Decision to Grant Accelerated Approval to Aducanumab for Alzheimer's Disease.

In June 2021, the US Food and Drug Administration (FDA) granted accelerated approval to aducanumab, a monoclonal antibody indicated for the treatment of Alzheimer's disease. The accelerated approval decision was controversial due to concerns about the use of an unvalidated surrogate measure, beta-amyloid, as the basis for approval and a lack of clinical outcome benefit. Between October 2021 and September 2022, we conducted a survey of a nationally representative group of internists, medical oncologists, and cardiologists to understand perspectives around aducanumab's approval and how this FDA

Current situation of pediatric clinical trials in China: focus on trials for drug marketing application and administrative approval.

BACKGROUND: Research and development of pediatric drug faces many difficulties and pediatric clinical trials remain a challenge. Since 2011, a series of measures have been taken to encourage research, development of drugs for pediatric patients in China. In this study, we analyzed pediatric clinical trials conducted in China to provide reference for research and development of pediatric drugs and formulation of relevant policies. METHODS: We conducted a cross-sectional observational study of pediatric trials registered in the Drug Trial Registration and Information Publication Platform before

Using Historical Data to Support Approval of Drug Products for Ophthalmia Neonatorum: Innovative Approach Toward Evidence-Based Medicine.

IMPORTANCE: Off-label treatment was common for ophthalmia neonatorum because only erythromycin ointment had been approved by the US Food and … fundamental elements used to evaluate whether extrapolation of effectiveness can be used to support drug approval.

Clinical Trial Evidence Supporting US Food and Drug Administration Approval of Novel Cancer Therapies Between 2000 and 2016.

IMPORTANCE: Clinical trial evidence used to support drug approval is typically the only information on benefits and harms that patients and clinicians can use for decision-making when novel cancer therapies become available. Various evaluations have raised concern about the uncertainty surrounding these data, and a systematic investigation of the available information on treatment outcomes for cancer drugs approved by the US Food and Drug Administration (FDA) is warranted. OBJECTIVE: To describe the clinical trial data available on treatment outcomes at the time of FDA approval of all novel c…

Simulation of nitrosamine drug substance-related impurities from secondary amine-related impurities in approved human drug products.

Nitrosamines, recognized as potential carcinogens, have emerged as impurities of concern in pharmaceutical products. This study evaluates the formation risk of active pharmaceutical ingredient (API) fragment nitrosamine drug substance-related impurities (NDSRIs) derived from secondary amine related impurities that may be present in human drug products throughout their shelf life. Leveraging publicly available data from the Food and Drug Administration (FDA) Global Substance Registration System (GSRS), the carcinogenic potency categorization approach (CPCA), and available maximum daily dose (MD

Identification and evaluation of repurposing Food and Drug Administration-approved drugs targeting the pE165R protein of African swine fever virus.

OBJECTIVE: African swine fever is a devastating swine disease lacking effective vaccines or antiviral drugs. The African swine fever virus (ASFV) pE165R deoxyuridine triphosphatase (dUTPase), critical for viral replication, is a promising antiviral target. Therefore, identifying inhibitors that target the pE165R protein represents a potential antiviral strategy. METHODS: The study computationally screened FDA-approved drugs against pE165R's crystal structure. Molecular dynamics simulations elucidated inhibitor-pE165R interactions, and in vitro enzymatic assays quantitatively assessed inhibito