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Pharmaxis’ Bronchitol Will Face Efficacy Questions At FDA Panel Review

 
• By Sue Sutter

Several statistical issues, including differential rates of missing data between treatment arms in the pivotal studies, make it difficult to assess mannitol’s benefit in cystic fibrosis patients, FDA says in briefing documents for the Pulmonary-Allergy Drugs Advisory Committee’s Jan. 30 meeting.

Pharmaxis Ltd. will need to overcome a host of statistical challenges to the efficacy of its cystic fibrosis drug Bronchitol (mannitol inhalation powder) to gain an FDA advisory committee’s endorsement Jan. 30.

In briefing documents released ahead of the Pulmonary-Allergy Drugs Advisory Committee’s meeting, FDA reviewers said their determination of mannitol’s efficacy...

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More from United States

Podium Policy Returns To US FDA, Or Is It Podcast Policy?

 
• By Michael McCaughan

US FDA critics have long decried the practice of using informal communications to convey regulatory expectations as podium policy. In the Makary era, the phrase may need to be updated because the agency now seems to be specializing in policy by podcast.

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• By Cathy Kelly

Tala Fakhouri said the agency’s use of the AI tool in the review process has generated lots of questions.

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• By Derrick Gingery

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Recent And Upcoming FDA Advisory Committee Meetings

 
• By Sue Sutter

Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.

More from North America

BIO Notebook: Policy Uncertainty Constraining Dealmaking

 
• By Mandy Jackson and Joseph Haas

Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 
• By Sarah Karlin-Smith

The former CDER director said she tells sponsors not to conduct an FDA-recommended study design or randomized trial if it will not work.

AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
• By Sue Sutter

Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.