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BrainStorm’s Phase III Data Do Not Support Clinical Benefit Of NurOwn In ALS, US FDA Says

 
• By Sue Sutter

Agency’s unusual public statement about an investigational product's development status may be aimed at countering bullish comments from the sponsor; FDA cites failure of pivotal trial on the primary and secondary endpoints and ‘modest excess in deaths’ with the cell therapy.

ALS On Chalkboard
The ALS patient community is eager for new treatments. • Source: Shutterstock

The US Food and Drug Administration’s unusual public statement about an investigational drug to treat ALS may be aimed at tamping down the sponsor’s bullish comments to the patient community about its Phase III data and prospects for regulatory approval.

On 2 March, the Center for Biologics Evaluation and Research quietly posted a statement on its website commenting on the...

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Bayesian Statistics In EU Clinical Trials: EMA Discusses Balancing Efficiency With Rigor

 
• By Eliza Slawther

Bayesian statistics could help clinical trial sponsors to include external information in their analyses, but concerns around bias and incorrect conclusions remain, the European Medicines Agency said.

Drug Sponsors Can Help Shape Agenda For FDA/EMA Pediatric ‘Cluster’ Calls

 
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US FDA’s ADHD Drug Relabeling Advances A MAHA Priority, Reviving Data Dispute

 
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How Will Regulatory Filing Winds Blow Globally For Elevidys Following Patient Deaths?

 
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Roche’s filing for Elevidys in the EU followed applications in Middle Eastern countries because those markets accept applications based on approval by the US Food and Drugs Administration.

More from Pathways & Standards

Drug Sponsors Can Help Shape Agenda For FDA/EMA Pediatric ‘Cluster’ Calls

 
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Companies can propose specific issues for discussion during the regulators’ monthly teleconferences, which focus on finding areas of agreement and reasons for nonalignment on specific pediatric development plans or general issues.

What Pharma Needs To Know About Vietnam’s Drug Registration Reforms

 
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Newly introduced rules for streamlining drug registration in Vietnam are said to present both opportunities and challenges for stakeholders.

England’s NHS Plan Eyes Cancer Vaccine Boost, Single Formulary & ‘Pay For Impact’ Drug Deal

 
• By Eliza Slawther

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