It is regrettable that the FDA issued a complete response letter to Jazz Pharmaceuticals for JZP-6. If there is a disease that can use some innovation, it’s fibromyalgia.
The CRL states that the FDA cannot approve the NDA in its present form. In the letter, the FDA discusses a number of topics, including the need for additional clinical studies, the appropriate patient population, methods for ensuring safe use, and the proposed REMS, concentration and trade name for the product.
Fibromyalgia has an interesting and unusual history. For years, fibromyalgia was not considered a “real” disease. Many physicians believed that fibromyalgia was a disease made up by pharmaceutical companies in order to sell more products. Today, the fibromyalgia “myth” is quite real. We now have a better understanding of the disease at a structural and genetic level. Other studies suggest there may be a genetic/hereditary component to the disease.
This brings us to Jazz and JZP-6. The active ingredient sodium butyrate, has been regrettably labelled the “date rape” drug due to its action as a CNS depressant with abuse potential. The drug is already on the market under the brand name Xyrem for the treatment of narcolepsy. To Jazz’ credit, the company appeared to anticipate and address the FDA’s concerns regarding REMS, limiting access to legitimate patients, and so forth.
I cannot say what the future holds for JZP-6. I do know that fibromyalgia therapeutic options, while effective, are limited. Hopefully, JZP-6 will see the light of day and help patients who suffer from this chronic, and very real disease.