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DUBLIN - Jazz Pharmaceuticals plc (NASDAQ:JAZZ), a $7.88 billion market cap pharmaceutical company with "GREAT" financial health according to InvestingPro metrics, announced Tuesday that Modeyso (dordaviprone) has been included in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines as a category 2A single-agent treatment option for patients with recurrent or progressive diffuse high-grade glioma harboring an H3 K27M mutation.
The NCCN recommendation comes shortly after Modeyso received accelerated approval from the U.S. Food and Drug Administration on August 6, 2025, for adult and pediatric patients aged one year and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy. The company maintains impressive gross profit margins of 92.14%, reflecting strong operational efficiency in its pharmaceutical development programs.
"The rapid addition of Modeyso to the NCCN Guidelines reflects the urgency of the unmet need that patients are faced with when diagnosed with this devastating and aggressive brain tumor," said Kelvin Tan, chief medical affairs officer of Jazz Pharmaceuticals.
Modeyso represents the first treatment option specifically for recurrent H3 K27M-mutant diffuse midline glioma, a rare and aggressive brain tumor primarily affecting the midline structures of the brain and spinal cord. Patients diagnosed with this type of glioma typically face a median survival of approximately one year from diagnosis and less than six months after disease progression following frontline therapy.
The FDA approval was based on an integrated efficacy analysis of 50 patients across five open-label clinical studies. The overall response rate was 22%, with a median duration of response of 10.3 months among responders.
Common adverse reactions reported in clinical trials included fatigue, headache, vomiting, nausea, and musculoskeletal pain. Serious adverse reactions occurred in 33% of patients.
Continued approval for this indication may be contingent upon verification of clinical benefit in the ongoing Phase 3 ACTION confirmatory trial, according to the company’s press release statement. While currently unprofitable, 11 analysts have revised their earnings upwards, and InvestingPro analysis suggests the company is undervalued with strong potential. For deeper insights into JAZZ’s valuation and growth prospects, including 12 additional ProTips and comprehensive financial analysis, investors can access the full Pro Research Report on InvestingPro.
In other recent news, Jazz Pharmaceuticals reported its second-quarter earnings for 2025, which revealed a larger-than-expected loss per share of -$8.25, compared to the anticipated -$7.62. Despite this, the company’s revenue slightly exceeded forecasts, reaching $1.05 billion. This earnings announcement comes alongside the official launch of Jazz Pharmaceuticals’ new drug, Modeyso, which targets a rare and aggressive form of pediatric brain tumor. Stifel reiterated its Buy rating on the company, maintaining a price target of $230, noting Modeyso’s potential to become the standard of care for this condition. Additionally, Truist Securities raised its price target for Jazz Pharmaceuticals to $205, maintaining a Buy rating, following a webcast on the commercial strategy for Modeyso. RBC Capital also increased its price target to $151, keeping an Outperform rating after an investor call about the new drug. These developments highlight the significant interest and potential seen by analysts in Jazz Pharmaceuticals’ recent activities.
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