Wednesday, September 18, 2013

At Biomarin Pharmaceutical, "Compassionate Use" meets Callous Disregard

At Biomarin, Compassionate Use meets Callous Disregard
Andrea Sloan on Huffington Post
Andrea Sloan interviewed by HuffPost
After a seven year battle against ovarian cancer Andrea Sloan’s oncologist advised her body cannot withstand any more chemotherapy and the only option for her is to get an investigational new drug known as BMN 673. Under most situations that would have not been overly complicated since the FDA approves of Compassionate Use in such cases where the patient is critically ill, has no other options, and could potentially benefit from the experimental drug.

But in order to seek FDA approval the sponsor, in this case Biomarin Pharmaceutical, will have to first agree to supply the drug. That is something they have so far not been willing to do.

BMN 673 is a powerful drug that inhibits enzymes responsible for repairing tumor DNA
proteins from functioning, thereby promoting cancer cell death. This family of drugs are known as poly ADP ribose polymerase or PARP inhibitors. According to a study published by scientists who may be employed at Biomarin, or may have an equity interest in the company BMN 673 is effective in much lower concentrations, selectively targets tumor cells with BRCA1, BRCA2, or PTEN gene defects, demonstrates more than 40% absolute oral bioavailability and exhibits a 200 fold greater potency than existing PARP inhibitors.

Given that kind of glowing success, one would think Biomarin would be more than willing to let Andrea try the drug. But while the company boasts of trial successes, executives appear to be focused on comparison with competing products, rather than the plight of a much deserving, and seriously ill patient who could die without this intervention.

In a letter obtained by KXAN TV, Biomarin CEO Jean-Jacques Bienaime in writing to Henry Fuchs, Chief medical Officer wants to refocus on competition while also asking about any clinical trials that might be recruiting. Apparently his email got threaded into a concerned shareholder’s query abut the Andrea Sloan case.

The competition he is referring to is quite likely Rucaparib, a Pfizer PARP inhibitor licensed to Clovis Oncology, which is in clinical trials currently in the United Kingdom. In the U.S. Clovis Oncology is conducting Phase I and II Rucaparib trials at several locations with The Sarah Canon Institute and several other Contract Research Organizations. The trial is registered with NHIS under identifier NCT01482715. I have linked this identifier to open the Clinical Trial page where contact information is available for each U.S. location to enroll in the trials.

Biomarin BMN 673 Cancer Drug Clinical Trial

Also while Biomarin might believe they do not have a clinical trial currently recruiting patients, they might want to correct the status of their trial NCT01286987 listed on the U.S. National Institutes of Health Clinical Trial platform where it clearly says “This study is currently recruiting participants.”

Accessed September 18, 2013.
The Huffington Post. "Texas Woman Makes Emotional Plea For Compassion As Drug Company Refuses Cancer Treatment." Accessed September 18, 2013.
KXAN. "BioMarin explains reasoning in Sloan case .com." Accessed September 18, 2013.
Clinical Cancer Research. "BMN 673, a Novel and Highly Potent PARP1/2 Inhibitor for the Treatment of Human Cancers with DNA Repair Deficiency." Accessed September 18, 2013.
Cancer Research UK: the UK's leading cancer charity : Cancer Research UK. "A trial looking at a new drug called rucaparib for breast and ovarian cancer, in people with BRCA gene faults (PH2/052) : Cancer Research UK : CancerHelp UK." Accessed September 18, 2013.
Home - "Study of BMN 673, a PARP Inhibitor, in Patients With Advanced or Recurrent Solid Tumors - Full Text View -"
Rucaparib Trials in the U.S.
Accessed September 18, 2013.

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