Biomarker guidance

FDA Guidance on Biomarker Qualification

Drug Development Tool, from CDER’s Biomarker Qualification Program. Provides qualified context of use (COU) for the biomarker plasma 29 fibrinogen for COPD trials, experimental conditions and constraints, patient selection, and options for enrichment designs. CDER Biomarker Qualification program

Storyline : Framework for scientific development and regulatory acceptance of biomarkers for use in drug development


SBIA WebinarEndtoEnd

Storyline : FDA encouraging End-to-End capture to streamline and standardize data capture and enhance FDA review


Oncologic Drugs Advisory Committee :  Necitumumab for Squamous Non Small Cell Lung Cancer, (Eli Lilly, IN)  

 An FDA advisory panel indicated  that the benefits of Eli Lilly’s investigational lung cancer drug necitumumab outweigh its risks. Most panel members described the drug’s survival benefit as modest, yet meaningful, and in line with other FDA-approved therapies, but they suggested measures should be adopted to mitigate its potential risks particularly potentially fatal thromboembolic events.

Storyline : Impact of ODAC favorable assessment on FDA Action and Labeling awaited.


FDA approved

PromusPROMUS Element Plus Everolimus-Eluting Platinum Chromium Coronary Stent System (Monorail and Over-The-Wire), and Promus PREMIER Everolimus-Eluting Platinum Chromium Coronary Stent System (Monorail and Over-The-Wire) (Boston Scientific Corporation , MN)

Regulatory Pathway : PMA Supplement to support expansion of the indications to medically treated diabetes. Original PMA approved Nov  2011

Indication (expanded) : The PROMUS Element™ Plus and Promus PREMIER™ Everolimus-Eluting Platinum Chromium Coronary Stent Systems are indicated for improving luminal diameter in patients, including those with diabetes mellitus, with symptomatic heart disease or documented silent ischemia due to de novo lesions in native coronary arteries ≥2.25 mm to ≤4.00 mm in diameter in lesions ≤34 mm in length.

PLATINUM Clinical Program (PMA and Supplements) : Several studies  – small vessel,  long lesion,  pharmacokinetics, quantitative coronary angiography, US Post Approval Study – latter for this supplement)

Effectiveness , The 12-month Cardiac Death (CD)/Myocardial Infarction (MI) rate +  diabetic endpoint of target vessel failure (TVF) rate – both significantly lower than performance goal

Safety : Adverse event rates  clinically acceptable. Death, MI, and stent thrombosis low.

Benefit/Risk Assessment : The risks and benefits of the PROMUS Element Plus similar to other approved drug-eluting stents (eg TAXUS). Probable benefits outweigh the probable risks, and adds to the treatment options available to patients with symptomatic coronary artery disease

entresto moreENTRESTO 

Regulatory Pathway : Priority Review (approved prior to Action Date), Fast Track designation

Indication : ENTRESTO is combination of sacubitril, a neprilysin inhibitor, and valsartan, an angiotensin II receptor blocker, indicated to reduce the risk of cardiovascular death and hospitalization for heart failure in patients with chronic heart failure (NYHA Class II-IV) and reduced ejection fraction.

Efficacy: Single pivotal study.  PARADIGM-HF demonstrated Entresto (sacubitril + RAS inhibitor valsartan) , superior to another RAS inhibitor (enalapril), in reducing the risk of the combined endpoint of cardiovascular death or hospitalization for heart failure, based on a time-to-event analysis (hazard ratio [HR]: 0.80,  p <0.0001). Also improved overall survival (HR 0.84; p = 0.0009)

 Safety: Angioedema higher with Entresto particularly in Black patients (2.4% vs 0.5%)

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