DUBLIN - Avadel Pharmaceuticals plc (NASDAQ:AVDL) announced that the U.S. Food and Drug Administration (FDA) has approved its once-at-bedtime narcolepsy medication, LUMRYZ, for pediatric patients aged 7 and older. This approval, granted on October 16, 2024, extends the use of LUMRYZ, which was previously available only to adults with narcolepsy, to a younger demographic facing the challenges of excessive daytime sleepiness (EDS) and cataplexy.
The approval of LUMRYZ for pediatric use marks an important development for children with narcolepsy and their families, as it offers a once-nightly treatment option without the need for a middle-of-the-night dose. The FDA's decision was supported by clinical trial results demonstrating the drug's efficacy and safety profile.
Greg Divis, CEO of Avadel Pharmaceuticals, emphasized the significance of this milestone for the company and the narcolepsy community, noting the transformational impact of LUMRYZ on patients' lives. Dr. Anne Marie Morse, a pediatric neurologist and sleep medicine specialist, also highlighted the benefits, stating that the single bedtime dose offers more options to patients and eases the treatment process.
Narcolepsy, a chronic neurological disorder affecting approximately 1 in 2,000 individuals in the U.S., can lead to EDS and cataplexy, which are sudden episodes of muscle weakness often triggered by strong emotions. The condition can significantly disrupt daily life and the sleep-wake cycle.
LUMRYZ, an extended-release formulation of sodium oxybate, was initially approved by the FDA on May 1, 2023, for adult patients. With its pediatric approval, the medication has also been granted Orphan Drug Exclusivity through October 16, 2031, providing Avadel with market exclusivity for this patient population for the next seven years.
The drug comes with a boxed warning due to its potential as a central nervous system depressant and for abuse and misuse. It is available only through a restricted program called the LUMRYZ REMS to mitigate these risks.
The most common adverse reactions reported in clinical trials for LUMRYZ include nausea, dizziness, enuresis, headache, and vomiting. The FDA's approval is based on the REST-ON trial, which showed significant improvements in EDS and cataplexy attacks compared to placebo.
Avadel Pharmaceuticals plc is dedicated to developing innovative medications that address treatment challenges. The company's commitment is reflected in the introduction of LUMRYZ to the market, offering a new treatment option for narcolepsy in both adults and now pediatric patients. This information is based on a press release statement.
In other recent news, Avadel Pharmaceuticals reported a net revenue of $41.5 million for the second quarter of 2024, driven by more than 1,900 patients using its narcolepsy treatment, LUMRYZ. The company has also launched a Phase 3 clinical trial for LUMRYZ in idiopathic hypersomnia and is awaiting an FDA decision for its use in pediatric narcolepsy. Despite Q2 operating expenses of $51.5 million, Avadel anticipates generating operating income in the third quarter and throughout the rest of the year.
Avadel received a favorable court ruling permitting the commercial launch of LUMRYZ for narcolepsy treatment, despite a motion by competitor Jazz Pharmaceuticals (NASDAQ:JAZZ) for an injunction that would have blocked its marketing. However, the court prohibited Avadel from seeking approval for an idiopathic hypersomnia (IH) indication for LUMRYZ until the expiration of a patent held by Jazz, a decision Avadel is currently appealing.
In a RESTORE study published in Sleep Medicine: X, 94% of participants favored the once-nightly narcolepsy medication, LUMRYZ, over traditional twice-nightly oxybate treatments. The FDA has granted LUMRYZ seven years of Orphan Drug Exclusivity based on its clinical superiority to existing treatments, particularly its once-nightly dosing regimen.
These developments are part of Avadel's recent efforts to expand its product portfolio and position in the pharmaceutical market. The company remains committed to closely collaborating with the FDA through the remainder of the review process for its supplemental New Drug Application (sNDA) for LUMRYZ.
InvestingPro Insights
The FDA approval of LUMRYZ for pediatric use is a significant milestone for Avadel Pharmaceuticals, potentially expanding its market reach and revenue streams. This development aligns with the InvestingPro Tip that analysts anticipate sales growth in the current year. The company's revenue has already shown remarkable growth, with InvestingPro data indicating a staggering 6260.23% increase in the last twelve months as of Q2 2024.
Another InvestingPro Tip highlights Avadel's impressive gross profit margins, which is reflected in the data showing a 94.62% gross profit margin for the same period. This strong profitability in its core operations could be crucial as the company scales up production and marketing for LUMRYZ's expanded use.
Despite these positive indicators, it's worth noting that Avadel is not currently profitable, as pointed out by another InvestingPro Tip. The company's operating income margin stands at -103.82%, suggesting that while revenue is growing, operational costs remain high. This is not uncommon for pharmaceutical companies in the growth phase, especially when launching new products.
For investors seeking a more comprehensive analysis, InvestingPro offers additional tips and insights. In fact, there are 5 more InvestingPro Tips available for Avadel Pharmaceuticals, which could provide valuable context for understanding the company's financial health and future prospects in light of this recent FDA approval.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.