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The rising impact of biomarkers in early clinical development

Drug Target Review

4 The definition has since evolved and expanded to include molecular signatures of pharmacodynamics and therapeutic response. As biomarkers become increasingly relevant in indicating the workings and effects of novel therapies, their potential as valuable clinical and regulatory endpoints is also gaining recognition. 21(10):3517.

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BioMarin Completes Acquisition of Inozyme Pharma

The Pharma Data

Strategic Expansion with INZ-701 The centerpiece of this acquisition is INZ-701, an investigational enzyme replacement therapy currently in advanced clinical development for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) Deficiency. per share.

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Defining Probability of Success in Clinical Trial Design with Commercial Software and R Coding

Cytel

Kyle Wathen, Vice President, Scientific Strategy & Innovation One of the pivotal metrics considered when designing a clinical trial is the study’s probability of success, which can be measured in several ways.

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Navigating Varying Regulatory Feedback in Clinical Oncology Trials: A Guide for Clinical Research Professionals

Conversations in Drug Development Trends

The clinical trials sector prompted a recent modernization of ICH E8 and a subsequent renovation of ICH E6 to provide updated guidance that is appropriate and flexible enough to address the increasing diversity of clinical trial designs and data sources employed to support regulatory and other health policy decisions.

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Why Proactive AME Studies are Critical to Accelerating Your Approval Journey

Conversations in Drug Development Trends

During clinical development, new chemical entities (NCEs) require an absorption, metabolism, and excretion (AME) study. This process involves a careful assessment of medical history and daily bowel habits.

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Overcoming Orphan Drug Development Challenges with Real-World Data and Evidence

PPD

While there is no universally accepted definition for rare disease, it is estimated that more than 10,000 distinct rare conditions exist, and people living with these rare diseases represent as much as 10% of the global population.

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FDA Issues “Cliffs Notes”-style Guidance on Cell and Gene Therapy; What Questions Did They Answer? (Part 1)

FDA Law Blog: Drug Discovery

Here, the draft guidance states that the appropriate timing for an INTERACT meeting should be when a sponsor has identified a specific product and has conducted some preliminary proof-of-concept (POC) studies but has not yet designed and conducted definitive toxicology studies.