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Wednesday 22 July 2026

FDA

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SCA meeting

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

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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PTC Therapeutics' CEO, Matthew Klein.

PTC pulls file for Duchenne therapy on FDA feedback

PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

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FDA says comparator dose scuppered Moderna's flu jab filing

FDA says comparator dose scuppered Moderna's flu jab filing

The FDA has defended its decision to issue a refusal-to-file (RTF) letter for Moderna's new mRNA-based influenza vaccine.

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Moderna cries foul as FDA blocks filing of mRNA flu shot

Moderna cries foul as FDA blocks filing of mRNA flu shot

The FDA won't accept Moderna's filing for an mRNA-based flu vaccine, in the latest signal of the changing regulatory climate for vaccines in the US.