Endpoints News
FDA rejects PTC’s rare disease drug Read in browser
Endpoints News
Thank you for reading, dupa dupackia!
basic
UPGRADE
M Tue W Th F
19 August, 2025
GLOBAL PHARMA AND BIOTECH SUMMIT - Embracing and industry reset
Biopharma is grappling with diverging policies between the US, EU, and Asia — including proposed drug pricing reforms in the US and new EU regulations taking effect this year. The need for strategic clarity has never been more urgent. Join us in London — in person and virtually — where we’re bringing together the industry’s most influential voices to chart a path forward.
top stories
1. HHS says ‘the science speaks for itself’ on mRNA cuts. Some of the scientists it cites disagree
2. FDA rejects PTC's Friedreich’s ataxia drug
3. Irish pharma exports to the US decrease after months of stockpiling
4. HHS seeks to dismiss Rhode Island lawsuit over reorganization plans
5. Roivant-backed VantAI and Halda line up $1B RIPTAC pact
6. Updated: Viking’s stock tanks as mid-stage obesity pill disappoints on efficacy, safety
7. George Church’s lab gets closer to creating human eggs in a dish, and a new startup plans to finish the job
8. CSL to spin out flu vaccine business, trim workforce in expansive overhaul
9. Exclusive: Sensorium raises $25M to test succulent-derived drug for anxiety
10. FDA delays decision on Regenxbio’s Hunter syndrome gene therapy by three months
more stories
 
Nicole DeFeudis
.

Two weeks ago, HHS announced that BARDA was canceling or reworking almost two dozen contracts for the development of mRNA vaccines. Endpoints News reporters reached out to authors from some of the studies that underpinned that decision. See what they had to say in Max Bayer's story here.

.
Nicole DeFeudis
Editor, Endpoints News
@Nicole_DeFeudis
HHS Secretary Robert F. Kennedy Jr. (AP Photo/John McDonnell)
1
by Max Bayer

Some of the stud­ies that un­der­pin the Trump ad­min­is­tra­tion’s de­ci­sion to veer away from mR­NA vac­cine re­search are be­ing mis­ap­plied, co-au­thors of the work say, mud­dling the move by HHS Sec­re­tary Robert F. Kennedy Jr. to back away from the tech­nol­o­gy.

End­points News reached out to au­thors rep­re­sent­ing more than 100 ci­ta­tions pro­vid­ed by the gov­ern­ment af­ter near­ly two dozen con­tracts re­lat­ed to mR­NA vac­cines were cut two weeks ago. At least a dozen au­thors said that HHS and Kennedy ei­ther mis­in­ter­pret­ed their find­ings out­right, or em­pha­sized that re­search in­to the plat­form should con­tin­ue. On­ly a cou­ple of re­searchers con­tact­ed by End­points ap­peared to sup­port the gov­ern­ment’s pull­back in fund­ing. Most did not re­spond when con­tact­ed about their re­search be­ing in­clud­ed and whether they felt it was be­ing ac­cu­rate­ly ap­plied.

Click here to continue reading
2
by Lei Lei Wu

The FDA re­ject­ed PTC Ther­a­peu­tics’ Friedre­ich’s atax­ia drug va­tiquinone, the com­pa­ny an­nounced Tues­day morn­ing.

The agency said that “sub­stan­tial ev­i­dence of ef­fi­ca­cy” was not shown for the Friedre­ich’s atax­ia ther­a­py, ac­cord­ing to PTC’s press re­lease. An ad­di­tion­al “ad­e­quate and well-con­trolled” study would be need­ed for a re­sub­mis­sion to the FDA.

Va­tiquinone failed its piv­otal study in Friedre­ich’s atax­ia in 2023, but the com­pa­ny point­ed to mea­sures that sug­gest­ed the ther­a­py could im­prove bal­ance and fa­tigue in ask­ing the FDA for ap­proval.

In Friedre­ich’s atax­ia, pa­tients lack the pro­tein fratax­in, which is key for the mi­to­chon­dria to func­tion prop­er­ly. Va­tiquinone is a small mol­e­cule drug that's de­signed to ad­dress that mi­to­chon­dr­i­al dys­func­tion and ox­ida­tive stress by block­ing an en­zyme called 15-lipoxy­ge­nase.

Click here to continue reading
2025'S BREAKOUT STARTUPS. WHO'S ON THE LIST? - Endpoints 11
Endpoints News returns to Boston’s State Room to announce the 2025 class of biotechs worth watching. Join the live unveiling and see the industry’s next chapter come into focus. Which biotechs will make the leap? Join us.
3
by Anna Brown

Ex­ports of chem­i­cal prod­ucts, which in­clude phar­ma goods, from Ire­land to the US dropped in June to the low­est they've been in a year, fol­low­ing months of drug stock­pil­ing by phar­ma com­pa­nies.

Ire­land ex­ports of chem­i­cals to the US de­creased to €3.2 bil­lion ($3.7 bil­lion) in June, com­pared to the €9.4 bil­lion ($11 bil­lion) re­port­ed in May, ac­cord­ing to Fri­day da­ta from Ire­land’s Cen­tral Sta­tis­tics Of­fice.

The de­crease fol­lows months of in­creased phar­ma im­ports in­to the US. In par­tic­u­lar, com­pa­nies rushed to stock­pile their US drug in­ven­to­ries in March to cush­ion any short-term tar­iff im­pact. In that month, Ire­land chem­i­cal ex­ports spiked to €23.9 bil­lion ($27.9 bil­lion). Pres­i­dent Don­ald Trump start­ed to threat­en phar­ma-spe­cif­ic tar­iffs in Feb­ru­ary.

While Ire­land-US phar­ma trad­ing pat­terns seemed to ease in April, ex­ports rose slight­ly again in May.

Click here to continue reading
4
by Nicole DeFeudis

HHS has asked a Rhode Is­land fed­er­al court to dis­miss a case brought by at­tor­neys gen­er­al from 19 states and Wash­ing­ton, DC, over the gov­ern­ment’s plan to re­struc­ture fed­er­al health agen­cies.

At­tor­neys for HHS on Fri­day ar­gued that “plain­tiffs’ claims fail on nu­mer­ous thresh­old grounds,” in­clud­ing that they lack stand­ing to chal­lenge the re­struc­tur­ing and re­duc­tions in force. HHS al­so said that “set­ting aside these thresh­old and ju­ris­dic­tion­al de­fects, plain­tiffs’ claims fail on the mer­its.”

“The de­part­ment has stat­ed that the re­struc­tured agency will con­tin­ue car­ry­ing out its statu­to­ry func­tions,” the HHS at­tor­neys wrote in the mo­tion. “Just be­cause it is not do­ing so in the way plain­tiffs pre­fer does not mean it is vi­o­lat­ing ap­plic­a­ble law.”

Click here to continue reading