DWQA QuestionsCategory: QuestionsBezafibrate 2
Ann asked 3 years ago
Hi,
This is a reply to your answer from 2 hours ago on bezafibrate.
First, thank you very much for your answer and your time. I would have a few other questions on bezafibrate:
Please note that I have been taking bezafibrate for 5 years as of now.

  1. You mentioned that bezafibrate has limited efficiency, does it mean that you wouldn’t recommend it? There seems to be quite a lot of controversy over the clinical results. To my knowledge, there were 2 clinical studies on bezafibrate on patients with lc-faod:
    1. A clinical study published in 2010 ‘Long-term follow-up of bezafibrate treatment in patients with the myopathic form of carnitine palmitoyltransferase 2 deficiency: The results suggest that BZ has a therapeutic effect in the muscular form of CPT2 deficiency.
    2. A second clinical study published in 2014: ‘Bezafibrate in skeletal muscle fatty acid oxidation disorders: a randomized clinical trial’ It provides class I evidence that that bezafibrate 200 mg 3 times daily is ineffective in improving changes in FAO and HR during exercise in adults with CPT II and VLCAD deficiencies.

The authors from these 2 clinical trials seem to be in contraction, and they have criticized each other’s methodology. I thought that this drug helped me reduce my symptoms. However, I am not sure if that was just a placebo effect. What’s your take on this? (I am also a bit worried about the safety profile of bezafibrate, which I’ve been taking for quite some time now…)

  1. Concerning the safety of REN 001: If I understand well, this drug is more selective on PPAR beta/delta. I’ve read that PPAR beta agonists can still cause system toxicity (cardiac hypertrophy, intestinal adenoma, liver fibrosis). Can ren001 still cause these side effects at the dose being studied in the clinical trials? What is the safety profile?

Thanks again for all your work and dedication!

Physician
replied 3 years ago

Ann, as you have noted the studies on bezafibrate are contradictory. I believe the second study was a stronger one and it correlates with my limited experience with bezafibrate use. However, since it is not approved in the US, i am unable to prescribe it routinely. Per my previous answer, PPAR delta agonists are far more specific in activating the clinically relevant metabolic pathways in the face of FAOD and in my published work are far more effective in restoring metabolic balance in FAOD cells that bezafibrate. REN001 is not a beta agonist and the safety concerns you question have not been seen with its use. I hope this helps.