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Trump's tax cut law expands orphan drug exclusions, could upend 340B program Read in browser
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7 July, 2025
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1. The Supreme Court passed on a state PBM case. Lawyers say a broader legal fight is brewing
2. Clinician groups sue RFK Jr. and HHS over Covid-19 vaccine recommendation changes
3. Trump's tax cut law expands orphan drug exclusions, could upend 340B program
4. FDA chief calls for 'meaningful' reduction of animal testing for new drugs
5. KalVista CEO says FDA review proceeded normally, was unaware of Makary's rejection ask
6. Despite delay, KalVista wins FDA approval after Makary's short-lived attempt to intervene
7. European Commission fines Alchem, closes API ‘cartel’ investigation 
8. Cogent’s mastocytosis drug hits ‘home run’ in registrational mid-stage trial
9. Compromised drug lot impacts Jasper's chronic hives study, stock falls
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Alexis Kramer
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The Supreme Court last week passed on a closely-watched case over state PBM regulation. But that won’t be the justices’ last opportunity to weigh in on the longstanding issue. Check out Nicole DeFeudis’ piece on how the timing for SCOTUS just wasn’t right this time around.

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Alexis Kramer
Editor, Endpoints News
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by Nicole DeFeudis

The Supreme Court re­cent­ly de­clined to wade in­to a fight over a state’s reg­u­la­tion of phar­ma­cy ben­e­fit man­agers. But le­gal ex­perts say it like­ly won’t be the last time the broad­er is­sue makes its way up to the high court.

The jus­tices left in place an ap­pel­late court de­ci­sion against cer­tain pro­vi­sions in an Ok­la­homa law that im­posed re­quire­ments on PBMs and their phar­ma­cy net­works. The case could have of­fered clar­i­ty on an is­sue that has trig­gered a wave of lit­i­ga­tion. But in­stead, low­er courts in oth­er cir­cuits are left to nav­i­gate a com­plex web of cas­es as states con­tin­ue to crack down on the en­ti­ties that have come un­der fire for their role in drug pric­ing.

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Manufacturing Day 2025
What are the new rules for biopharma manufacturing? The Trump administration’s tariff policies have thrown the world economic order off its axis, and manufacturing is squarely in the middle of the upheaval. Join us as we break down what’s really happening behind the scenes — get your spot.
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by Max Bayer

A group of or­ga­ni­za­tions rep­re­sent­ing thou­sands of doc­tors na­tion­wide sued HHS Sec­re­tary Robert F. Kennedy Jr. over his de­ci­sion to change Covid-19 vac­cine rec­om­men­da­tions for kids and preg­nant women.

The fed­er­al law­suit filed Mon­day in Mass­a­chu­setts is led by the Amer­i­can Acad­e­my of Pe­di­atrics and al­leges that Kennedy's ac­tion “im­me­di­ate­ly ex­pos­es these vul­ner­a­ble pop­u­la­tions to a se­ri­ous ill­ness with po­ten­tial­ly ir­re­versible long-term ef­fects and, in some cas­es, death.”

“This is not a hy­po­thet­i­cal con­cern, but a press­ing pub­lic health emer­gency that de­mands im­me­di­ate le­gal ac­tion and cor­rec­tion,” ac­cord­ing to the com­plaint. The plain­tiffs are look­ing to over­turn Kennedy’s di­rec­tive that in­sti­tut­ed the change.

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President Donald Trump after signing the 'One Big Beautiful Bill' on the South Lawn at the White House (Yuri Gripas/Abaca/Sipa USA/Sipa via AP Images)
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by Zachary Brennan

Pres­i­dent Don­ald Trump on Ju­ly 4 signed in­to law his wide-rang­ing tax cuts and rec­on­cil­i­a­tion pack­age, in­clud­ing an ex­pan­sion of or­phan drug pro­tec­tions from Medicare ne­go­ti­a­tions, and near­ly $1 tril­lion in Med­ic­aid cuts that could up­end the op­er­a­tions of hos­pi­tals that pro­vide cheap­er drugs and care un­der the 340B fed­er­al pro­gram.

The or­phan drug pro­vi­sion is a sig­nif­i­cant win for the drug in­dus­try. Un­der the law, which is for­mal­ly called the One Big Beau­ti­ful Bill Act, any or­phan or rare dis­ease drugs, po­ten­tial­ly in­clud­ing block­busters with mul­ti­ple or­phan in­di­ca­tions, won't face Medicare price ne­go­ti­a­tions un­der the In­fla­tion Re­duc­tion Act.

Nei­ther of the first two rounds of drugs se­lect­ed for ne­go­ti­a­tion in­clud­ed or­phan drugs. But the phar­ma in­dus­try raised con­cerns that the way the IRA was writ­ten could lim­it the ex­clu­sion to on­ly drugs with one rare dis­ease in­di­ca­tion.

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FDA Commissioner Marty Makary (Photo by Aaron Schwartz/Sipa USA via AP Images)
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by Zachary Brennan

FDA Com­mis­sion­er Mar­ty Makary pledged to "mean­ing­ful­ly re­duce" un­nec­es­sary an­i­mal test­ing that's cur­rent­ly used to bring new drugs to mar­ket and to test them in ear­ly de­vel­op­men­tal stages.

While Makary's pro­pos­al is light on specifics, a re­duc­tion of an­i­mal test­ing could low­er R&D costs, speed up drug de­vel­op­ment, and "po­ten­tial­ly even trans­late in­to low­er drug prices," Makary said at an FDA-NIH work­shop at the FDA's head­quar­ters on Mon­day.

"One of our biggest goals is to re­duce an­i­mal test­ing in a way that's mean­ing­ful and con­tin­ues to pro­tect against pub­lic safe­ty," he added.

Makary's FDA in April said an­i­mal test­ing re­quire­ments, which vary de­pend­ing on the type of drug in de­vel­op­ment, will be "re­duced, re­fined, or po­ten­tial­ly re­placed" via "a range of ap­proach­es, in­clud­ing AI-based com­pu­ta­tion­al mod­els of tox­i­c­i­ty and cell lines and organoid tox­i­c­i­ty test­ing in a lab­o­ra­to­ry set­ting."

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Ben Palleiko, KalVista Pharmaceuticals CEO