FDA

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New FDA knockback hits uniQure and Huntington patients hard

uniQure looks like it may have to run another trial of its Huntington's disease gene therapy AMT-130, delaying the highly anticipated programme.

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US lawmakers take FDA to task over rare diseases record

Lawmakers and patient advocates in the US are concerned that the FDA's regulation of rare disease therapies has been inconsistent and overly cautious.

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FDA maps new regulatory route for ultra-rare diseases

The FDA has sketched out its 'plausible mechanism' regulatory pathway for individualised therapies that treat ultra-rare diseases.

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Scrabble lettering of Two and One on grass

Makary says FDA will "end two-trial dogma" for approvals

The FDA plans to adopt a new policy that will do away with the default requirement to base regulatory filings for new medicines on two pivotal trials.

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