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Introduction

Due to the recent success of RedHill Biopharma’s Stage 3 trial of RHB-104, more patients and doctors have become interested in Atypical Mycobacterial Antibiotic Therapy  (AMAT) as a treatment option for chronic immune conditions, such as Crohn’s disease and multiple sclerosis. RHB-104 contains a triple combination of antibiotics that showed significant effectiveness in Crohn’s disease, and includes clarithromycin, Rifabutin and clofazimine. While clarithromycin and Rifabutin are easily available worldwide, clofazimine is not.

Limited Availability of Clofazimine

Clofazimine was first developed by researchers at Trinity College in Dublin, Ireland. It’s primary use is for the treatment of leprosy. It is also used to treat nontuberculous mycobacterial infections. With its inclusion in RHB-104, there developed a growing niche for the drug in Crohn’s disease. But this medication is not readily available at pharmacies or chemists, because few cases of leprosy exist in the Western world.

Clofazimine has been classified as an orphan drug, which means that few companies produce the medication due to limited potential for profitability. It does not mean that the drug is illegal, or has been banned. A community member has kindly provided Human Para with a variety of options he considered on his quest to legally obtain clofazimine.

 

Option 1:  FDA SPIND (Single Patient Investigational New Drug) via Expanded Access

In recent years, the United State Food & Drug Administration (FDA) has taken a more lenient approach in allowing doctors and their patients to make decisions about treatments that have not yet received full FDA approval for a specified condition. This practice is known as Expanded Access, or Compassionate Use. While clofazimine is FDA approved for leprosy, it is not yet approved for use in Crohn’s disease. Prescribing an FDA approved drug  for a secondary, unapproved condition is considered an “off label” use of that medication, and can be a legal gray area for doctors. If approval is granted, the FDA will issue a SPIND # to the investigating physician, for use with the patient named in the protocol.

In parallel, Novartis, the Swiss pharmaceutical manufacturer of clofazimine, has developed its own Managed Access Program. Through Managed Access, Novartis is able to work directly with physicians (but not patients) to guide them thru the necessary regulatory steps. Once the SPIND # is assigned by the FDA, they arrange access for your clinic to clofazimine.

In our case, our gastroenterologist developed her plan/protocol, based on the RedHill MAPUS Clinical Trial protocol and input provided by Prof. Borody. The University required signature approval of several of colleagues, which she received. The request was also approved by the Institutional Review Board (IRB).

Following IRB approval, our doctor submitted the package, along with a few other completed forms, to the FDA for approval under Expanded Access. This was a relatively straightforward process. From the time the protocol was developed it took 4-6 weeks to get IRB signatures, and FDA approval.

Novartis required hard copies (not email) of the various approvals, after which they arranged shipment of the clofazimine directly to the clinic. The approach is sometimes seen as a “trial of n=1.”

Option 2: Prof. Thomas Borody, Centre for Digestive Diseases in Sydney, Australia

Professor Borody’s clinic in Sydney has a relationship with a compounding pharmacy for providing clofazimine to Crohn’s disease patients worldwide. By contacting their office, your physician can work with the staff to complete case histories, prescriptions, and arrange 90-day shipments, at a fixed cost. NOTE: Your physician must contact the CDD directly via email, phone or fax. Due to the volume received, inquiries from patients cannot be answered.

Option 3:  Order Online from Canadian Pharmacies

A Google search for clofazimine will reveal dozens of offers from Canadian online pharmacies, promising to provide clofazimine to United States patients, worry free. While there are hundreds of such online pharmacies, the (supposedly) most reliable have certification or approval from two agencies: CIPA and Pharmacy Checker.

CIPA, the Canadian International Pharmacy Association, has authorized 66 Canadian websites to display its seal, which sites are licensed and regulated by the government for safety.

Pharmacy Checker has a verification program where they check the licensing and industry standards of applicants to assure they are in good standing in the countries in which they are located. Unlike CIPA, Pharmacy Checker is open to pharmacies around the world. Pharmacies in certain countries are subject to onsite inspection.

We looked at every online pharmacy that had either CIPA approval, Pharmacy Checker approval, or both. Of more than 80 sites checked, only 8 offered clofazimine. Some of these sites appeared to be owned by the same person. A few of those that offered clofazimine accepted credit card payments, but some only accepted a check or payment via Paypal. This could be a consideration, and a possible red flag. The pharmacies we checked stated they received their supply from India. China is also another possible supplier.

While the easiest of the three options above, supply chain and verification methods may be an issue for some patients. Additionally, for Canadian patients, these online pharmacies are not available since they are (ironically) prohibited from shipping to Canadian residents. Lastly, any international mail order medication is subject to customs inspection, which may delay delivery.

Thank you to Kerry Schuster for sharing his journey and providing our community with this resource.