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EMA And FDA Compared – The Case Of Minoryx’s Leriglitazone

 
• By Francesca Bruce

The EMA offers more flexibility than the US FDA when it comes to missed primary endpoints in certain circumstances, meaning a potentially quicker route to authorization in the EU.

Pathway
EU and US regulators take a different approach to Minoryx's orphan drug leriglitazone • Source: Shutterstock

Spanish biotech firm Minoryx Therapeutics hopes a newly approved Phase III trial will lead to the US approval of its orphan drug leriglitazone for X-linked adrenoleukodystrophy (X-ALD) in 2026 at the earliest.

Meanwhile, the European Medicine Agency’s review of the drug is well under way, with a decision on marketing authorization expected at the end of 2023 or the beginning of 2024

Key Takeaways

  • The EMA offers a more flexible approach than the FDA when primary endpoints are missed for orphan products where there has been little natural history data of the disease to inform trial design.

  • The review of leriglitazone is more advanced in Europe than in the US

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