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When is a Confirmatory Trial “Underway” or Conducted with “Due Diligence” Enough for Accelerated Approval? FDA Explains Its New Authorities

FDA Law Blog: Drug Discovery

However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.

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The rising impact of biomarkers in early clinical development

Drug Target Review

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.

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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).

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Merck’s WINREVAIR Succeeds in Phase 3 Trial for Pulmonary Arterial Hypertension

The Pharma Data

These findings represent a pivotal advancement in the treatment of this rare, progressive, and life-threatening disease. Participants were classified as WHO Group 1 PAH, functional class (FC) II or III, indicating moderate to severe disease at either intermediate or high risk of clinical deterioration.

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Nerandomilast

New Drug Approvals

Clinical Trials: Phase 3 clinical trials have shown that nerandomilast can slow lung function decline in patients with IPF and PPF. Efficacy: The trials demonstrated that nerandomilast led to a smaller decline in forced vital capacity (FVC), a measure of lung function, compared to placebo. SCHEME 1H NMR (400 MHz, DMSO-D6)  1.57–1.84

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Future-proofing drug development with GenAI

Drug Target Review

It is becoming increasingly evident that generative artificial intelligence (GenAI) is a resourceful tool for helping pharmaceutical companies reduce manual tasks required by clinical trials. This is especially relevant with today’s heavier focus on enhancing personalised medicine via broader emerging scientific findings.

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Amgen Unveils Phase 2 Obesity Trial Results for Monthly Maritide at ADA’s 85th Scientific Sessions

The Pharma Data

Phase 2 Study: Transformative Weight Loss and Glycemic Control The Phase 2 trial focused on evaluating the efficacy and safety of MariTide in individuals with obesity, both with and without Type 2 diabetes (T2D). The data revealed compelling weight loss outcomes , particularly when compared to placebo.

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