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Full Pediatric Labeling Is Lacking For One Third Of Orphan Drugs, US FDA Finds

 
• By Bridget Silverman

The past three years have seen an increase in number of orphan drug labels containing appropriate pediatric information.

The US Food and Drug Administration’s efforts to improve drug labeling for pediatric patients have been making solid gains in orphan drugs, with the number of orphan products approved with full pediatric labeling hitting a peak in 2015-2018, according to an agency report to Congress.

The FDA analyzed 20 years of orphan drug approvals for the report, which was required by the 2017 FDA...

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More from Approvals

Japan Gives Global-First Recommendations To Taisho’s Vornorexant, Santen’s Sepetaprost

 
• By Lisa Takagi

Japan gives world-first approval recommendations to two domestically originated drugs, for insomnia and glaucoma/ocular hypertension.

New EU Filings

 
• By Neena Brizmohun

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Sanofi Secures EU Fast-Track Status For ‘Game-Changing’ Sleeping Sickness Drug

 
• By Neena Brizmohun

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EU 2025 Novel Drug Approvals See Conditional Authorizations Outpacing Previous Years

 
• By Neena Brizmohun

The six EU conditional marketing authorizations granted in the first half of 2025 alone equal the full count for 2024 and come close to the annual totals for the previous two years. Meanwhile, 1H 2025 saw a 30% rise in new active substance approvals compared to the same period last year.

More from Product Reviews

New EU Filings

 
• By Neena Brizmohun

Arimoclomol, Zevra Therapeutics's treatment for Niemann-Pick disease Type C, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.

Sanofi Secures EU Fast-Track Status For ‘Game-Changing’ Sleeping Sickness Drug

 
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Repurposed Generics For Rare Diseases: Janet Woodcock Proposes Shortcut

 
• By Grace Moser

A Duke-Margolis working group is developing ideas to enable non-profit firms to seek US FDA approval for new indications for off-patent medicines. The former acting commissioner thinks citizen petitions might be the fastest route.