Q&A: Approved drugs versus supplements

To the RCDP community, we want to address the on-going confusion over the safety and regulatory requirements for developing approved drugs versus "supplements”. Although we have gone in-depth on these differences in the past (see our video on this topic), we wanted to be straight up on the key issues.

Our program at Med-Life has always been focused on a scientifically-credible and regulatory-compliant drug development process. We have always tried to be as available as possible to the community, answering questions about drug development and the status of the program. We have noticed that there are a few questions that frequently seem to come back up within the community. We thought now would be a great time to answer those questions in a format that everyone can refer back to. Below are a few of the most commonly asked questions, but if you have others, please feel free to reach out to us through our Facebook or Instagram accounts.

Is MLD developing a drug or supplement?

MLD is developing a compound called PPI-1011, that while based on naturally occurring molecules, is a non-natural structure. That makes it something called a new chemical entity. Drugs that are new chemical entities, and/or synthetically manufactured products, require an extensive and formal safety and efficacy evaluation based on FDA rules. In fact, anything that is designed to treat, mitigate, prevent, or cure a disease is considered a drug. That means anything being advanced as a legitimate treatment for RCDP would be bound by the regulatory authorities, regardless of the structure of the compound.

Can’t plasmalogen precursors be considered natural products? Wouldn’t that mean they don’t need regulatory approval?

Any “natural product” on the market, regardless of its manufacturing process, which is administered above what can normally be obtained in the diet absolutely requires formal regulatory approval and an NDI (new dietary ingredient) designation. Any products on the market with dosage label claims that are higher than what can be obtained from natural dietary sources are considered unapproved, adulterated illegal products. Anyone who says otherwise is completely naive about medicinal product and drug development regulations and are encouraged to watch our detailed video.

What about other plasmalogen products?

We fully appreciate that there are numerous and various “plasmalogen” supplements on the market. What anyone decides to use on their own children is their decision, and we as a company will never stand in the way of that decision. However, we have yet to see ANY credible and/or compelling evidence of clinical safety or efficacy for any plasmalogen supplement, for ANY indication, and we follow the plasmalogen literature closely and have openly posted interesting summaries through over 100 plasmalogen blog posts. With our current understanding of plasmalogen pharmacokinetics, manufacturing requirements, dosing, and safety, it is clear that “supplements” currently do not have the purity or concentrations needed for clinical benefit. In our recently published peer-reviewed Phase I paper, we demonstrate why PPI-1011 is far superior to any other available product, and that “the data on supplements to date indicate that they are clinically unviable due to limited concentration and potency.”

And furthermore, an N of one participant in a study, is not a study. Any credible scientist obviously knows this, but we live in crazy times where unfortunately unproven and false claims about products have become the norm.

 What has MLD done to advance an RCDP Therapy?

Our goal from day one has been to develop an active and safe plasmalogen-based therapeutic product that could be officially evaluated through an approved clinical trial in RCDP patients. MLD is not interested whatsoever in sidestepping drug approval regulations. Millions have been invested to date into GMP-grade drug manufacturing, GLP preclinical safety studies in numerous species, a first-in-human Phase I clinical trial, and extensive plasmalogen research and development (for example, building a new genetic animal model from scratch). We have also assembled one of the strongest rare-disease regulatory teams one could wish for. We have done it right so far and will continue to do so.

 Why is the process taking so long? Isn’t MLD committed to a treatment?

We have always been completely transparent and have never “hidden” anything from anyone. We put our trials on clinical trials.gov, we publish our findings in peer-reviewed journals, and we constantly interact with the RCDP community. We’re doing this the proper way for the benefit of RCDP patients and the community, so that there is a long-term safe and viable treatment option for years to come. This commitment was echoed by participants of the PFDD last summer who made it clear that they are only interested in novel treatment options that have a demonstrated safety profile. Obviously, we would always prefer faster timelines on the entire process, but the reality of drug development is that the required studies simply take time. Sourcing animals takes time. Planning trials takes time. Meeting with vendors and regulatory agencies takes time. Finding investment takes time. Just because it is taking time does not mean things aren’t progressing.

With that we will close by giving a huge shout out to the entire RCDP community for continuing to support the program, travelling to Nemours, and partaking in our events. We as a small company are here for you, and of course you can reach out to any of us at anytime with questions. As you know, we absolutely love to talk about the program and RCDP! Hope to see you at Rhizokids next summer!