from the Newsletter of the Multidisciplinary Association for Psychedelic Studies
MAPS - Volume 3 Number 4 Winter 1992-93


MDMA Patentability and Orphan Drug Designation
Rick Doblin


Why in the world would MAPS hire a patent attorney? After all, MDMA was patented in 1914 in Germany by the Merck Pharmaceutical Company. That patent has long since expired leaving MDMA in the public domain. If MDMA could be legally manufactured no payments to Merck would be required. The answer is that there are several kinds of patents for molecules; composition of matter, manufacturing process, and use patents. Even if the composition of matter patent has expired, it is still possible to gain legal control over the use of a particular molecule for a particular purpose through obtaining a use patent or through patenting an inexpensive and efficient manufacturing process. While the composition of matter patent for MDMA has expired and excellent manufacturing processes have been published in the scientific literature, I wasn't so sure about the legal possibilities for a use patent.

The issue of use patents for MDMA is very timely now that human studies can begin. MAPS has opened a Drug Master File for MDMA at the FDA and is preparing to sponsor FDA-approved MDMA research. If the research goes extraordinarily well, MAPS may one day have the opportunity and responsibility to manufacture and market MDMA to appropriately trained and licensed physicians. It could be a major setback if MAPS funded MDMA research and secured approval for its medical use only to have someone else control it through an inexpensively obtained use patent.

I decided to hire a patent attorney to try and insure that MDMA's potential medical use would not be bottled up by anyone else's use patent. I also wanted to explore whether MAPS might obtain such a use permit itself. Howard Lotsof, the founder of NDA International, a for-profit corporation which has obtained several use patents for ibogaine in the treatment of heroin and cocaine addiction and for the amelioration of the symptoms of narcotic withdrawal, referred me to his patent attorneys, Miskin & Mandelbaum of New York. I found them to be both genuinely interested in MAPS' unusual question and expertly qualified to addess it.

My first hope, obviously, was that no use patents for MDMA were in existence. Less obviously, I hoped that there was already too much written about MDMA's therapeutic potential for anyone, MAPS included, to patent its use. The goal of MAPS is to facilitate research with psychedelics and to help develop their medical uses, not to corner any market. If any well-funded pharmaceutical company thinks they can profit from MDMA by sponsoring research and gaining FDA approval before MAPS does, I want them to feel free to step right in and do so. However, if MAPS does succeed in securing FDA approval to market MDMA for some clinical indication, sales might generate some financial return which could be used for further research consistent with our non-profit agenda. Perhaps MAPS could even become self-funding, a possibility that will certainly be a relief to those of you whom I repeatedly ask for additional donations.

As I hoped and expected, there are no current use patents for MDMA. Attorney Ursula Day of Miskin & Mandelbaum had the following to say about the chances that MAPS, or anyone else, could obtain such a patent:

"Without the benefit of more exhaustive background research on the therapeutic uses of MDMA, we are of the opinion that it would be rather difficult for someone to obtain patent protection on such use of MDMA. While there is always a possibility that somebody might wish to file such an application unbeknownst to you, an applicant in this situation would have to overcome any other possible prior art which the Examiner might have available. You are correct in pointing out that published references to prior use would indeed be a bar to obtaining a patent for the therapeutic use of MDMA. However, in the event that an applicant were to obtain a patent on the therapeutic use, there is a great likelihood that the patent would not withstand an attack based on invalidity... Although the search we conducted was far from exhaustive, it appears from the few references that you have submitted to us that there is sufficient publication on the therapeutic uses and a variety of indications and applications for MDMA that would prevent another from obtaining patent protection on a method".

Hence it is unlikely that anyone can get use patents for MDMA. But that is not the end of the story.

Orphan Drug Designation

It still might be possible to obtain a seven year exclusive right to market MDMA for a particular clinical indication under the sponsorship of the FDA's Orphan Drug Office. When a drug is for a rare disease, occuring to fewer than 200,000 people a year, or when for whatever reason the drug developer cannot make its money back in seven years, the financial incentives are usually not sufficient for a pharmaceutical company to develop a drug. Congress created the FDA's Orphan Drug Products Office to change the set of incentives facing drug developers so that Orphan Drugs would be developed. These incentives include protocol assistance, tax credits for investors, and most importantly, if the FDA ever determines that MDMA is safe and effective for some clearly defined clinical condition, a seven year exclusive right to market the drug for that clinical indication. Furthermore, these incentives are available to profit-making pharmaceutical companies, non-profit organizations, educational institutions, or private individuals.

For a real-life example of just how this works, consider drug development in the field of the medical use of marijauna. The FDA has already approved an oral THC pill (Marinol) but limited its use to nausea control in cancer chemotherapy patients. Unimed, the company that markets Marinol, got intrigued by the possibility that THC might be helpful in stimulating appetite in AIDS patients. They were able to make a credible case to the FDA's Orphan Drug Products Office that oral THC for the treatment of the HIV-related wasting syndrome deserved Orphan Drug designation. For the last several years they have been gathering data which supports the appetite stimulating properties of oral THC. They are conducting Phase 3 trials and seem not all that far away from being able to market Marinol for the wasting syndrome, maybe even within 1993.

From what I can tell, an excellent case can be made that MDMA in the treatment of pain in end-stage cancer patients deserves Orphan Drug designation on financial grounds. Over the next several months, I will be exploring these issues further with the Office of Orphan Products Development.