– Zhenya Senyak
Sometimes it starts with zebras grazing placidly under the African sun while nearby a pride of lions tears into one of their own, newly brought down. This time they escaped claw and fang. This time they’re OK. They go back to living.
What choice do they have after all?
The Sanofi story starts that way because we hear about death of one of our own on a clinical trial and feel impotent. What can we do about it?
It doesn’t take long to realize the source of frustration and anger.
By the time we head toward a clinical trial we’ve run out of options. We rely on the good intentions, the professionalism and honesty of physicians and medical institutions, the oversight of the FDA. Some guys are good, some aren’t.
Because we’re weak and in pain, because we need this chance for relief, for a cure, we let ourselves forget clinical trials are part of a multi-billion dollar industry. In that often predatory system, we can become just the fodder fed in at one end to produce blockbuster drugs at the other. Or not.
They can write off their losses. We can’t.
We have to change the way things are done.
One of us died, at least four of us were seriously injured in the Sanofi Phase III trial of Fedratinib (SAR302503), the old TargeGen Jak2 inhibitor. The trial was suddenly put on hold and three days later, Sanofi packs up its bags and folds all further development.
What of the 400 or so patients on trial? Patient counseling? We’re told to get in touch with our hematologists. Make sure they check our thiamine levels.
For an explanation we’re fed corporate gobbledygook: “Following a risk benefit analysis…Sanofi determined the risk to patient safety outweighed the benefit fedratinib would bring to patients.”
How many ways is that statement bullshit? What, for example, is the risk to patient safety in getting booted out of a trial with no notice, no options, no counseling? And if the risk, based on the minimal information we’ve been permitted to learn, is based on a thiamine deficiency, vitamin B1, isn’t that something easily treated? Watch the Claire Harrison video and hear it for yourself.
The only thing about that Corporate PR statement I believe is that Sanofi did actually undertake a risk benefit analysis the weekend of November 16 and it weighed its own risk against its own benefits. With its weak oncology reputation further tattered by these reports, what chance does it have to come up against Jakafi?
Maybe worse than the cold calculations of a distant corporation, is the failure of our own government, the FDA, not only to monitor effectively but to release information to us, the patients, the guinea pigs lining up for a crippling bolus of drugs. After Sanofi refused to answer simple questions – how many of us injured? where did it take place, why didn’t you address the problem by screening patients and providing supplements instead of folding — we turned to the FDA.
Our request for information was denied . We filed a Freedom of Information Act request and the FDA’s FOIA group denied that as well on the grounds – get this – that it violates trade secrecy! Our appeal gets bumped up another bureaucratic notch to the OGIS and the Department of Justice.
We have to change the way things are done. Patients need open, honest communications with principal investigators. We need a seat at the table when clinical trial results are being evaluated.
Researchers don’t discuss procedures, risks, injuries, results with us any more than they would with laboratory guinea pigs and knock-out mice. Their job is to follow the trial protocols, report any severe adverse events not to us but to the study sponsor.
180 million reasons for Sanofi to quit.
But why did Sanofi come this far with the highly successful JAKARTA trials and fold right at the Phase III finish line, with all the FDA paperwork for an NDA done, just weeks from filing and almost certain approval?
So far we don’t know. But our investigation and leads from inside sources might provide a clue. 180 million clues.
Sanofi bought TargeGen three years ago for $560 million…to be doled out as the drug reached various milestones. Reportedly, $30 million was recently paid to the original investors for achieving Phase III end points. What remained were two payments of $90 million each, due upon receipt of FDA and EHA approval. That’s $180 million that won’t have to be paid now that that the trial is closed and further development cancelled.
For giant Sanofi, with sales of 35 billion euros or so a year, it might not be a compelling reason on its own but put in the balance with bad publicity and uncertainty about the root issues attached to the drug, it just adds 180 million other arguments on the side of closing down the clinical trial.
And so far as those patients benefitting from the drug?
Ah, that’s too bad.
What can we do?
Our first step is to form a taskforce, draft a petition and get it signed by thousands of blood cancer patients, a petition asking that clinical trial investigators be included under the new Sunshine Act that’s part of the Affordable Care Act. It’s a beginning. We need to pressure the FDA to be more responsive to patients. This is an election year, if we organize we can change things.
If you can participate send an e-mail to ourMPNforum@gmail.com. No message necessary. In the SUBJECT AREA type Zebra.
We’ll know what you mean.
Chronology of abandonment
Thursday, November 14: A patient told MPNforum she received notice from Sanofi to discontinue fedratinib.
Friday, November 15: A Sanofi spokesman calls MPNforum to alert patients patients that a “temporary clinical hold” had been placed on the drug due to four cases of Wernicke’s encephalopathy, one fatal. Sanofi would issue a press release on Monday and would make medical experts available via teleconference.
Monday, November 18: Sanofi pulls the plug on all fedratinib clinical trials.
November 21 Sanofi responded to three questions:
MPNforum: As SAR302503 (fedratinib) bears many similarities with ruxolitinib (Jakafi) what work is being done to investigate the difference that might have produced these events.
SANOFI: Sanofi has completed a detailed review of each patient with Wernicke’s like symptoms. The underlying cause is unknown. The cause is thought to be multi-factorial. Sanofi is collaborating with the steering committee and investigators to present and publish the results of the trials. Sanofi has contacted other companies who are engaged in the development of JAK2 therapies to share information on the cases in the interest of protecting patient safety and supporting new therapies
MPNforum: Why pull the drug instead of addressing the toxicity issue (See Dr. Claire Harrison video, above, on managing thiamine (vitamin B1) deficiency?
SANOFI: Sanofi has determined that the potential risk to patients being treated with Fedratinib outweighs the potential benefits. While the company recognizes that many patients have derived individual benefit as participants in the clinical trials, the safety of patients is paramount, and the potential risk has led to this difficult decision. Patients should consult with their physicians to seek alternative treatment as appropriate.
MPNforum: How many severe adverse events happened and at what centers did they occur.
SANOFI: Because our investigation and analysis of safety signals is ongoing, we are not releasing specific numbers.
How we were blindsided:
MPNforum Headline: Fedratinib (SAR-302503): Clinical hold on Sanofi trial, Patients to stop taking drug Story in TSR here
Take me back to the Contents
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