MOBIO Member Highlight
Avadel Pharmaceuticals, with U.S. headquarters in St. Louis, MO: working to transform the lives of people with narcolepsy
Avadel is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options.
Our current lead drug candidate, LUMRYZ™ (sodium oxybate) for extended-release oral suspension was first conceived by an employee having a loved one with narcolepsy, a chronic neurological disorder in which the brain can’t regulate sleep-wake cycles. Based upon our heritage of drug delivery technology, the mission to bring an extended-release formulation of sodium oxybate to patients living with this disorder has been underway for approximately 10 years.
Sodium oxybate has been studied in the treatment of narcolepsy since the 1970s. Immediate-release sodium oxybate first received FDA approval in 2002 for the treatment of narcolepsy and is recognized by many as the standard of care treatment for this chronic condition. However, the only commercially available formulations require an unusual dosing regimen: patients take one dose at bedtime, typically falling asleep in 5-15 minutes, and then must wake up, 2.5 to 4 hours later, to take their second dose of medication. This dosing regimen is counterintuitive for people with a sleep disorder and creates additional challenges for patients already suffering from disrupted night time sleep.
Sodium oxybate is a difficult drug moiety to apply traditional extended-release technology to; however, the propriety drug delivery technology platform from Avadel enabled us to develop a much-needed solution to the twice-nightly dosing regimen.
In Avadel’s REST-ON clinical trial, once-at-bedtime LUMRYZ demonstrated statistically significant and clinically meaningful improvements across all co-primary endpoints, specifically improving the amount of time that patients could stay awake, an overall improvement as assessed by their clinicians, and a reduction in cataplexy, which is sudden muscle weakness that people with Narcolepsy Type 1 experience, typically in response to an emotion, such as laughter or surprise. Cataplexy may be as profound as full-body collapse, or may be more subtle, such as dropping items or slurred speech.
In July 2022, LUMRYZ received FDA Tentative Approval for the treatment of cataplexy or excessive daytime sleepiness in adults with narcolepsy. FDA Tentative Approval means that a medication has demonstrated efficacy, safety, and manufacturing quality. However, due to a patent that is listed in the FDA’s Orange Book, a final approval decision cannot be given until after disposition of the patent. In this regard, the US District Court for the District of Delaware recently ruled that the patent blocking a final approval decision for LUMRYZ was improperly listed and ordered the patent removed from the FDA’s Orange Book. Upon removal of that patent from the Orange Book, Avadel will be able to request a transition from Tentative Approval to Final FDA Approval.
Our delay in final FDA approval has not deterred us from our mission: we know patients are desperate for an option that will not require a forced, middle-of-the-night awakening, and they motivate us to ensure we urgently bring LUMRYZ to the narcolepsy community.
Our Values are key to how we will fulfill this promise; at Avadel, it was imperative that these are not simply printed words, but that they imbue how we will serve patients – with LUMRYZ and beyond.
In everything we do, we consider the patient experience, listening to their needs as we pursue the development and delivery of medicines that will have the most meaningful impact on those we serve.
We live the Avadel values so we can be the best for our patients, our community, and each other.
We are relentless in our pursuit of solutions to address the challenges patients face with current treatment options. We have the tenacity and burning desire to overcome obstacles and never give up. We act with a sense of urgency because we understand that patients shouldn’t wait for innovative solutions. We passionately believe in what we are doing and whom we are doing it for – the patient.
· Confidence with Humility
We are confident in our ability to recognize and overcome the challenges before us, yet we know when to ask for support. We are confident in our individual expertise, yet humble enough to know we don't have all the answers. We push each other to be our best and treat each other with respect.
We recognize that it takes courage to make the bold decisions that can have the greatest impact for patients. We ask the tough questions because if not us, then who? Our diverse team sees our challenges as our biggest opportunities and continually pushes outside of their comfort zones to drive meaningful progress.
· Insights to Impact
We listen to our stakeholders, use data to drive decisions, and act on what we learn. We use insights to identify opportunities and take actions that deliver the greatest impact for patients. Our efforts are thoughtful and evidence-based, resulting from a deep understanding of what it takes to effect meaningful change for patients and those who care for them.
We strive to create a level of connectivity that goes beyond working together. Rooted in the trust we earn every day, our team is inclusive, valuing diverse perspectives and work every day to lift each other up in pursuit of improving the lives of the patients we serve. This translates into one team driving success for each employee, success for the company, and most importantly, success for every patient.
We are proud of our values, of our unique offering based upon our propriety technology, and the substantial unmet need that once-at-bedtime LUMRYZ will fulfill, if final FDA approval is granted. Avadel is also proud to have our U.S. office in Missouri; as we expand, we look to harness talent in the biotechnology industry to be part of our mission to best serve patients.