For 20 years, Jazz has been focused on ensuring patients have innovative treatments for rare, hard-to-treat disorders. We have done this in part by expanding the science behind existing options to create new or better standards of care in challenging diseases such as narcolepsy, idiopathic hypersomnia, and other sleep disorders.
Patient safety is a key tenet of our mission. Unfortunately, a recent action by the U.S. Food and Drug Administration (“FDA”) could compromise patient safety and undermine appropriate patient care by disregarding scientific data supporting the benefits of low sodium, which is why we have taken legal action against FDA in U.S. Federal Court.
We believe FDA acted outside its authority under the Orphan Drug Act when it disregarded the orphan-drug exclusivity protecting our product Xywav®, a low-sodium oxybate, and approved LumryzTM, a high-sodium oxybate and extended-release reformulation of Jazz’s older product, Xyrem®. Xywav contains 92% less sodium than either Xyrem or Lumryz and FDA previously recognized that the significant reduction in chronic sodium burden achieved by Xywav will have clinically meaningful benefits in a substantial portion of narcolepsy patients by reducing their risk for hypertension and other cardiovascular comorbidities. FDA recognized that reduced sodium oxybate would be safer than higher sodium oxybate and awarded seven years of orphan-drug exclusivity to Xywav, which does not expire until 2027.
Unfortunately, FDA disregarded their own previous decision and long-standing position on the importance of lowering sodium intake as well as our earned exclusivity and approved Lumryz. As explained in our court filings, we believe FDA acted inconsistently with the Orphan Drug Act, in implementing its regulations and applying longstanding agency policy. FDA also appears to reverse prior decisions by its own staff and experts, without a reasoned justification.
Our most important concern, however, is that FDA’s reasoning is scientifically unsupportable. There is no data suggesting that Lumryz is more effective than Xyrem or Xywav at improving sleep architecture or reducing the disrupted nighttime sleep (“DNS”) experienced by narcolepsy patients. No head-to-head trials have been conducted, and separate studies have produced evidence demonstrating positive impacts on sleep quality and sleep architecture that are broadly similar for each of the three oxybate products. Critically, there is no data supporting FDA’s statements that once-nightly dosing can normalize the sleep architecture of adults with narcolepsy or that twice-nightly dosing is somehow harmful to narcolepsy treatment.
In contrast, FDA’s conclusion about the medical benefit of Xywav’s reduced sodium burden is well grounded in scientific consensus. At a 9 g nightly dose, Xywav reduces the sodium burden of any sodium oxybate product (including Lumryz) by more than 1,500 mg every night, which is a clinically meaningful reduction according to the American Heart Association (AHA), the National Academies of Sciences, Engineering, and Medicine (NASEM), the U.S. Department of Agriculture (USDA), the U.S. Department of Health and Human Services (HHS), and public health authorities around the world.
We are concerned that by implying there is a positive therapeutic impact of once-nightly dosing and minimizing the established benefit of reduced sodium, FDA’s decision may undermine appropriate patient care. Patients may be swayed by the false hope, fostered by FDA’s decision, that Lumryz will return them to normal sleep, but they will experience higher cardiovascular risk due to elevated nightly sodium.
Separately, Jazz believes that the proper functioning of the Orphan Drug Act is critical to ensuring the ongoing development of products to treat rare diseases by providing meaningful incentives for companies to continue to innovate on treatments for these patients. Protecting innovation is important to the ongoing research and development of therapies for people living with these serious, debilitating conditions.
Jazz employees work tirelessly each day to help address the needs of people living with devastating diseases, and it is crucial that the laws and policies related to drug approvals support companies in making the needed investments that lead to new, more effective and safer treatments in order to bring patients options that help them live healthier and more fulfilled lives.
