Pow­er­ful Dems or­ches­trat­ed cam­paign at FDA to pro­vide Bio­gen drug to a dy­ing, high-pro­file fundrais­er

Back in 2008, Dal­las tri­al lawyer and high-pro­file De­mo­c­ra­t­ic Par­ty fundrais­er Fred Baron was dy­ing of mul­ti­ple myelo­ma. Des­per­ate to gain ac­cess to Bio­gen’s Tysabri, an MS drug then be­ing test­ed for myelo­ma in ear­ly-stage stud­ies, his wife Lisa Blue Baron reached out to the com­pa­ny but ran di­rect­ly in­to a brick wall of re­sis­tance.

“[Bio­gen] won’t ap­prove for fred be­cause he is too sick and if it fails him, it could skew the out­come of the tri­als(this told to me by his wife),” not­ed a fam­i­ly friend in the er­ror-rid­den post in­clud­ed in the batch of hacked emails from John Podes­ta — the chair­man of Hillary Clin­ton’s pres­i­den­tial cam­paign — dumped by Wik­iLeaks on Sat­ur­day.

In­censed, Baron’s wife reached out to in­flu­en­tial friends in the De­mo­c­ra­t­ic Par­ty.

If I am un­able to get drug I will take out full pg add in boston globe to ceo and board mem­bers.vFred is run­ning out of time. He’s so sick and needs this drug. Thanks so much.

In short or­der, a whole line­up of high-pro­file of­fi­cials rang­ing from for­mer Pres­i­dent Bill Clin­ton to Sen­a­tor John Ker­ry and then FDA Com­mis­sion­er An­drew von Es­chen­bach were di­rect­ly lob­by­ing for­mer Bio­gen CEO Jim Mullen to pro­vide the drug for com­pas­sion­ate use.

One seg­ment of the Podes­ta email ex­change in­cludes:

I got through to Mullen.  He had al­ready had ex­ten­sive con­ver­sa­tions with Sen­a­tor Ker­ry and Lance Arm­strong and had heard from Pres­i­dent Clin­ton, too.  The an­swer he gave in every case was [no].  I be­lieve that I un­der­stood it cor­rect­ly that Ker­ry had or­ga­nized a con­fer­ence call with Mullen and FDA Com­mis­sion­er Von Es­chen­bach.  I hear that Von Es­chen­bach said it was fine with him for the drug to be used in Fred’s case.  How­ev­er, Bio­gen be­lieves that Von E does not have that au­thor­i­ty giv­en the strict FDA con­di­tions on the use of the drug.

And in the next sec­tion:

I just had a dis­ap­point­ing call with Dr. Von Es­chen­bach. FDA had a phone con­fer­ence with the med­ical and sci­ence of­fi­cers from Bio­gen. FDA told them there would be no prej­u­dice to the clin­i­cal tri­al if this drug were used for Fred. They were ap­pre­cia­tive but gave no in­di­ca­tion they would change their po­si­tion, which is that a cor­po­rate de­ci­sion has been made they would not go out­side the clin­i­cal tri­al. I am re­al­ly up­set about this, per­son­al­ly, since FDA has been great to help. I have not had the time to­day to reach out to some on the board of Bio­gen, but hope to do so yet to­day. I felt last night, af­ter talk­ing with Andy von Es­h­en­bach, that with clear­ance from FDA, the com­pa­ny would be ok.

Look­ing back over Luke Tim­mer­man’s cov­er­age at the time for Xcon­o­my, it’s in­ter­est­ing to note that Baron got the drug be­cause the FDA and the Mayo Clin­ic pro­vid­ed it to him af­ter the agency found a le­gal ba­sis to do so in an emer­gency sit­u­a­tion.

Bio­gen, though, had con­tin­ued to in­sist that any use of Tysabri for myelo­ma in Baron’s case could cre­ate a sit­u­a­tion that would cause reg­u­la­tors to re­strict its use. This was two years af­ter the FDA had al­lowed Bio­gen and Elan to put the drug back on the mar­ket af­ter it was pulled fol­low­ing the death of sev­er­al pa­tients due to PML.

The de­bate over ac­cess to drugs un­der com­pas­sion­ate use poli­cies has helped spur a whole se­ries of “Right to Try” laws around the coun­try. For many pa­tients, it can be a frus­trat­ing ex­er­cise, try­ing to con­vince a bio­phar­ma com­pa­ny to pro­vide a drug to them in their last dy­ing days. For many biotechs, though, com­pas­sion­ate use re­mains a vex­ing is­sue as well. These pro­grams can be ex­pen­sive and al­so rais­es the threat that an un­ex­pect­ed ad­verse event can force a com­pa­ny to sus­pend work on a drug.

Fred Baron got the drug, but there was no hap­py end­ing. The be­hind-the-scenes strug­gle end­ed with his death on Oc­to­ber 30, 2008, soon af­ter the con­tro­ver­sy erupt­ed.

Bio­mark­er 'roadmap­s' and the fu­ture of can­cer R&D; Cur­tain rais­es on #AS­CO22; Pfiz­er, No­var­tis tack­le drug ac­cess; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

While this was not a week for earth-shattering news, there were certainly a lot of interesting tidbits. If you found this recap helpful, please recommend it to your friends and colleagues. We’ll see you on the other side of the long weekend.

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Keep­ing pres­sure on Am­gen, Mi­rati draws mixed re­views on lat­est cut of KRAS da­ta

As the close runner-up to Amgen’s Lumakras in the KRAS race, any data cut from Mirati’s adagrasib continues to draw scrutiny from analysts. And the latest batch of numbers from ASCO is a decidedly mixed bag.

While a quick comparison suggests that adagrasib spurred slightly more responses and led to a longer overall survival than Lumakras among a group of non-small cell lung cancer patients, its duration of response appears shorter and the safety profile continues to spark concern.

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Ann is one of ViiV Healthcare's newest spokespeople as the retired school administrator speaks up about her HIV status.

GSK's Vi­iV de­buts next evo­lu­tion in HIV med Dova­to cam­paign with new spokes­peo­ple and new mes­sage

When Ann saw the first TV commercials for HIV medicine Dovato, she didn’t see herself represented. So the 74-year-old retired school administrator who’s been living with HIV since 1998, reached out to GSK’s ViiV Healthcare and asked why not?

Now Ann is one of three people starring in ViiV’s latest Dovato campaign called “Detect This.” The next-step evolution in the branded campaign plays on the word “detect” — often used in describing HIV status under control as undetectable — but in this case, uses the word as a directive for people to understand they can use fewer medicines.

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Switzer­land to de­stroy over 600,000 ex­pired dos­es of Mod­er­na Covid vac­cine

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

Lina Khan, FTC chair (Graeme Jennings/Pool via AP Images)

Pile-on over PBMs con­tin­ues with FTC com­ments and a new bi­par­ti­san Sen­ate bill

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

Pharma brands are losing their shine with US consumers who are now thinking about the economy and inflation instead of Covid. (Credit: Shutterstock)

Phar­ma brands fade in an­nu­al Har­ris con­sumer vis­i­bil­i­ty poll: Mod­er­na drops off and Pfiz­er dips

As Covid-19 concerns are fading in the US, so is biopharma visibility. The annual Axios Harris Poll survey to determine and rank the 100 most top-of-mind brands in the US finds Moderna, which was No. 3 last year, not on the list at all for 2022, and Pfizer sinking 37 spots.

However, it’s not that Moderna or Pfizer did anything wrong, it’s just that Americans have moved on to other worries beyond Covid.

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HHS Secretary Xavier Becerra (Jacquelyn Martin/AP Images)

HHS fin­ish­es off Trump-era rule that would've erased ba­sic FDA regs with­out fre­quent re­views

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.

Tran­si­tion to new Eu­ro­pean clin­i­cal tri­als in­fo sys­tem starts slow­ly

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

Vi­iV Health­care looks to make long-act­ing HIV pre­ven­tion shot ac­ces­si­ble in low- and mid­dle-in­come coun­tries

The Joint United Nations Programme on HIV and AIDS set a lofty goal back in 2019 to end the HIV epidemic by 2030. But according to the World Health Organization, infection rates are not falling rapidly enough to meet that target.

GSK’s ViiV Healthcare thinks it can help change that.

On Friday, ViiV announced that it’s in talks with the UN-backed Medicines Patent Pool (MPP) for patent rights to its cabotegravir long-acting HIV injectable for pre-exposure prophylaxis (PrEP) in low- and middle-income countries.