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FDA rolls out new rare disease framework Read in browser
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14 October, 2026
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top stories
1. Sanofi's new R&D chief says Western pharma shouldn't ignore China's role in drug pipelines
2. New FDA framework favors consistency over flexibility on rare disease drugs
3. Verastem makes its stand as a RevMed competitor in pancreatic cancer
4. Vir makes solid tumor T cell engager deal with Gilead
5. Arrowhead’s brain-targeting drug lowers Alzheimer’s tau protein in early study
6.
news briefing
Ultragenyx's second PRV sale; Biomea's oral GLP-1 data leads to stock slump
Kevin Miller
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Western drugmakers who ignore China’s innovations and burgeoning pipeline do so at their peril, Sanofi’s new R&D chief, Paulo Fontoura, tells our Maggie Fick. The upshot: There's little room for a not-invented-here mindset.

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Kevin Miller
Deputy Editor, Endpoints News
1
by Maggie G Fick

Sanofi's new R&D chief said on Wednes­day that West­ern drug­mak­ers can­not ig­nore Chi­na's ac­cel­er­at­ing in­no­va­tion and need to con­sid­er how the coun­try’s emer­gence as a...

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2
by Max Bayer

Then-act­ing FDA lead­ers said over the sum­mer that reg­u­la­to­ry flex­i­bil­i­ty was­n't the stan­dard they were striv­ing for when the agency gauges rare dis­ease drugs.

Now...

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Health Tech Day 2026
The entire healthcare ecosystem is more Silicon Valley-ified than ever before. What tech has actually changed? Is biopharma behind? Find out, join us for Health Tech day.
3
by Lei Lei Wu

Ve­rastem On­col­o­gy had a big Wednes­day morn­ing: It an­nounced new da­ta amid a tense com­pe­ti­tion for the next-wave KRAS agents, as well as a $250...

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4
by Lei Lei Wu

Vir Biotech­nol­o­gy and its CEO Mar­i­anne De Backer have notched yet an­oth­er deal, this time with Gilead.

Gilead will pay Vir $65 mil­lion up­front, with an­oth­er...

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5
by Ryan Cross

Ar­row­head Phar­ma­ceu­ti­cals has shown that a ge­net­ic med­i­cine ad­min­is­tered as a sub­cu­ta­neous in­jec­tion can seem­ing­ly trav­el in­to the brain and low­er lev­els of tau, a pro­tein...

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News Briefing: Quick hits from the biopharma web
6
by ENDPOINTS

Plus news about Unit­ed Ther­a­peu­tics, Liq­uidia, Sanofi, Pharm­ing, Sky­hook Bio and Al­tay Ther­a­peu­tics.

Ul­tragenyx Phar­ma­ceu­ti­cal sells sec­ond PRV this month: The com­mer­cial-stage rare dis­ease drug­mak­er...

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