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Ultrangenyx's Sanfilippo syndrome therapy approved Read in browser
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17 September, 2026
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1. Exclusive: Internal CMS email confirms Globe and Guard exemption strategy
2. FDA approves Ultragenyx's gene therapy for Sanfilippo syndrome type A
3. Bristol Myers scraps $100M Orum cancer drug after reviewing Phase 1 data
4. The realities of China’s prolific biotech rise are felt at FDA
5. UK government committees call for probe into US pharma deal
6. Lilly-backed Orbis does up to $1.4B macrocycle deal with Novo
7. Roche turns to Dualitas to find new bispecific approaches for immunology
8. Bavarian Nordic names Tarja Stenvall as new CEO
9. Roche eyes label expansion for Lunsumio after confirmatory lymphoma trial win
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Max Bayer
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FDA leaders acknowledged the difficulty in assessing and reviewing data from China's increasingly dominant biotech sector when they spoke with Endpoints News on Tuesday. I wrote about the agency's clear-eyed take and how it can reliably move forward.

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Max Bayer
Senior Reporter, Endpoints News
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by Max Bayer

The Cen­ters for Medicare and Med­ic­aid Ser­vices has ex­empt­ed drug­mak­ers from two pric­ing demon­stra­tion pro­grams in Medicare if they agree to pro­vide ad­di­tion­al re­bates in...

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by Max Gelman

The FDA grant­ed ac­cel­er­at­ed ap­proval to a rare dis­ease gene ther­a­py from Ul­tragenyx on Thurs­day, giv­ing the com­pa­ny a much-need­ed boost in an oth­er­wise dif­fi­cult...

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by Kyle LaHucik

Bris­tol My­ers Squibb has end­ed de­vel­op­ment of a twist on the pop­u­lar an­ti­body-drug con­ju­gate field.

The Phase 1 as­set that was axed is BMS-986497/ORM-6151, ac­cord­ing...

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by Max Bayer

WASH­ING­TON — What do you do when an eco­nom­ic ri­val swift­ly be­comes a crit­i­cal play­er in the in­dus­try you reg­u­late?

The FDA is com­ing to...

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by Anna Brown

Six UK gov­ern­ment com­mit­tees have called for an in­ves­ti­ga­tion in­to the cost of the UK’s phar­ma­ceu­ti­cal trade deal with the US.

The let­ter, pub­lished...

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