Dear Jean-Jacques Bienaimé,
As a study patient and participant in the BMN701 Pompe Program, I am horrified and stunned to learn of the sudden discontinuation of this study by BioMarin, which was communicated by a simple notification dated June 22, 2016. The reasons for the discontinuation were, according to BioMarin itself, neither danger to the patient nor ineffectiveness of the medication.
This occurred after our supporting physicians from the Villa Metabolica at Mainz University Hospital had assured us in recent weeks that the study results were promising and discontinuation of the study was unlikely. I must point out that I do not reproach the study physicians with anything, as they were just as surprised by discontinuation of the study as their patients.
The reasons for discontinuation of the study: “Management announced that it is focusing on products worth more than $500M and that BMN-701 represented a $400 million opportunity.”
In other words, the product is profitable, but is to be discarded because the profit is not high enough.
“Another reason cited by the company was the presence of another treatment for Pompe disorder.”
BioMarin was surely aware that an approved medication for Pompe disease already existed before the start of the study because approval was granted as early as 2006.
“Continued development of BMN-701 might have also proved expensive for BioMarin in light of the company’s 2017 guidance for non-GAAP profitability, analyst Alethia Young pointed out. The company is now seeking a partner to bring the asset into a Phase II trial.
“We had previously given the company full economic credit for development. Now, we are adjusting to give BioMarin a 20% royalty on peak sales of ~$350M for BMN-701,” Young wrote. The probability of success of the treatment has also been lowered from 65 percent to 50 percent.
(All English quotations in italics are taken from an article published on the Website bezinga.com by Monica Gerson dated June 13, 2016.) Link to the article:
After the prospects for the study results were praised as positive in January, see:
further development of the medication is apparently too costly for the investors/BioMarin.
The consequence of this abrupt development is that no study medication has been available in Germany since the end of July 2016 and the therapeutic successes achieved are beginning to wane. For me, for example, BioMarin’s decision means that the measured improvement of my pulmonary function achieved by the study medication will worsen again. The distance I can walk in the 6-minute walking test has improved by almost 40 meters over the course of the study. I stand more stably and muscle pain and uncontrolled muscular spasms have disappeared. I am generally stronger and do not become exhausted so quickly, which is important as I still work for a living. Now, I am forced to seek an alternative treatment to maintain my remaining muscle power, which in my case is demonstrably less effective than the study medication.
All therapeutic successes will be lost because BioMarin strictly rules out “compassionate use.”
Patients, such as myself, who put themselves at the service of BioMarin to develop a promising medication and who have stayed the course during the time-consuming study procedure, are just dropped like a hot potato. Without any consideration for the consequential damage to health and the psychological strain caused by the decision by the pharmaceutical company, BioMarin refuses all contact. The company does not even see any need to inform its patients (a relatively small group) about the decision personally.
This behavior dishonors the company and destroys all credibility for future studies. Any potential candidate for a study in the future will think twice about serving such an unreliable pharmaceutical company.
This makes a mockery of the claim in your LinkedIn profile as Chairman and CEO to care about economic empowerment, human rights, and science and technology.
Unfortunately, it seems there is scant hope of reconsideration. This is not the first time the behavior of BioMarin has sacrificed Article 3 („Everyone has the right to life, liberty and security of person“) of the Universal Declaration of Human Rights to economic interests, despite the self-description of its CEO. Study patients of BioMarin in countries in which treatment with the alternative enzyme replacement therapy with Myozyme is not approved, could now also lose their lives sooner.
Eschborn, September 3, 2016