Latest news with #JazzPharmaceuticals
Medscape
4 days ago
- Business
- Medscape
FDA Approves Drug for Aggressive Glioma
The FDA has granted accelerated approval to dordaviprone (Modeyso, Jazz Pharmaceuticals) for H3 K27M-mutant diffuse glioma, marking the first time a systemic therapy has been approved for the aggressive glioma subtype. H3 K27M-mutant diffuse glioma is a grade 4 midline tumor that primarily affects children and young adults. Median survival is about a year. Historically, treatment has been limited to radiation, with few options available after recurrence besides palliative care. Dordaviprone is a first-in-class oral imipridone that disrupts tumor cell metabolism and increases tumor susceptibility to immune-mediated attack. Jazz Pharmaceuticals bought Chimerix in early 2025 for almost $1 billion based on dordaviprone's commercial prospects. 'Dordaviprone has the potential to rapidly become a standard of care for a rare oncology disease and also contribute durable revenue beginning in the near-term,' Jazz CEO Bruce Cozadd, said in a press release at the time. Approval was based on a pooled analysis of five single-arm studies enrolling 46 adults and four children with recurrent tumors previously treated with radiation, according to a statement from the FDA. Adults received 625 mg once weekly while pediatric patients received lower, weight-adjusted weekly doses. Mutation status was confirmed by immunohistochemistry or sequencing of archival tumor tissue. The overall response rate was 22%. The median time to response was 8.3 months, and the median duration of response (DOR) was 10.3 months. Among 11 patients with objective responses, 73% had a DOR of at least 6 months, and 27% had a DOR of at least 12 months. Seven of 15 evaluable patients (46.7%) were able to have corticosteroid dose reductions of at least 50%, and performance status improved in six of 34 (20.6%) patients. Sixty percent of subjects experienced treatment-related, treatment-emergent adverse events (TR-TEAEs), including fatigue (34%), nausea (18%), decreased lymphocyte levels (14%). The most common serious adverse events were hydrocephalus (8%) and nausea (8%). Grade 3 TR-TEAEs occurred in 20%, most commonly fatigue (10%). One case each of seizure and pulmonary embolism were seen. There were no treatment-related deaths. The drug's prescribing information includes warnings and precautions for hypersensitivity, QTc interval prolongation, and embryo-fetal toxicity, according to the FDA statement. A phase 3 trial is underway to confirm the benefit of dordaviprone for recurrent H3 K27M-mutant diffuse glioma. M. Alexander Otto is a physician assistant with a master's degree in medical science and a journalism degree from Newhouse. He is an award-winning medical journalist who worked for several major news outlets before joining Medscape. Alex is also an MIT Knight Science Journalism fellow. Email: aotto@
Yahoo
4 days ago
- Business
- Yahoo
US FDA approves Jazz Pharma's drug for rare brain tumor
By Padmanabhan Ananthan and Puyaan Singh (Reuters) -The U.S. Food and Drug Administration said on Wednesday that it has approved Jazz Pharmaceuticals' drug to treat diffuse midline glioma, a rare and aggressive tumor, in adults and children aged one year and older. The approval expands Jazz's cancer drug portfolio beyond its existing treatments for certain blood and lung cancers. This is the first FDA-approved systemic therapy for diffuse midline glioma with a specific mutation that has progressed despite prior treatments, the agency said. The drug, Modeyso, is expected to become available in the U.S. in the coming weeks and is administered as a once-weekly oral capsule. Diffuse midline glioma (DMG) is a rare and aggressive brain tumor that primarily affects children and young adults. It develops in the brain's and spinal cord's midline structures, such as the brainstem, thalamus, and spinal cord. An estimated 3,940 people are living with this tumor in the United States, according to NIH data. The FDA decision was based on data from 50 patients in five clinical studies, showing that the drug helped shrink tumors in about 22% of cases. Among those who responded, the benefit lasted a median of just over 10 months. "We think it fits very well in terms of addressing a very high unmet need," said Rob Iannone, Chief Medical Officer at Jazz Pharmaceuticals, ahead of the decision. Jazz Pharmaceuticals acquired the drug in March through its $935 million purchase of Chimerix. The company says it plans to work with doctors and advocates to make the treatment available as quickly as possible. Solve the daily Crossword
Reuters
4 days ago
- Health
- Reuters
US FDA approves Jazz Pharma's drug for rare brain tumor
Aug 6 (Reuters) - The U.S. Food and Drug Administration said on Wednesday that it has approved Jazz Pharmaceuticals' (JAZZ.O), opens new tab drug to treat diffuse midline glioma, a rare and aggressive tumor, in adults and children aged one year and older. This marks the first FDA approval of a systemic therapy for diffuse midline glioma with a specific mutation that has progressed after previous treatments, the agency said. Diffuse midline glioma (DMG) is a rare and aggressive brain tumor that primarily affects children and young adults. It develops in the brain's and spinal cord's midline structures, such as the brainstem, thalamus, and spinal cord. Jazz Pharmaceuticals acquired the drug in March through its $935 million purchase of Chimerix.
Yahoo
5 days ago
- Business
- Yahoo
Jazz Pharmaceuticals PLC (JAZZ) Q2 2025 Earnings Call Highlights: Strong Neuroscience ...
Total Revenue: $1.05 billion in Q2 2025, a 2% increase year-over-year. Xywav Revenue: $415 million, a 13% increase year-over-year. Epidiolex Revenue: $252 million, a 2% increase year-over-year. Rylaze Revenue: $101 million, a 7% decrease year-over-year. Zepzelca Revenue: $75 million, an 8% decrease year-over-year. Ziihera Revenue: $6 million in Q2 2025. Operating Cash Flow: $519 million in the first half of 2025. Cash and Investments: $1.7 billion at the end of Q2 2025. Adjusted Net Loss: $505 million, driven by a $905 million non-tax deductible acquired IPR&D charge. Warning! GuruFocus has detected 6 Warning Signs with JAZZ. Release Date: August 05, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Positive Points Jazz Pharmaceuticals PLC (NASDAQ:JAZZ) reported over $1 billion in total revenue for Q2 2025, driven by strong performance in its neuroscience assets. Xywav sales increased by 13% year-over-year, with significant net patient additions in both narcolepsy and idiopathic hypersomnia. Epidiolex continues to show strong demand and is expected to reach blockbuster status this year. The company is optimistic about the potential approval of dordaviprone and Zepzelca, which could drive future growth. Jazz Pharmaceuticals PLC (NASDAQ:JAZZ) has a robust pipeline with promising data from ongoing clinical trials, including Zepzelca and zanidatamab. Negative Points The oncology portfolio faced near-term headwinds, with a 1% decrease in total oncology product sales compared to Q2 2024. Rylaze sales decreased by 7% year-over-year due to changes in pediatric ALL treatment protocols. Zepzelca sales decreased by 8% year-over-year, impacted by increased competition and changes in treatment patterns. The company revised its financial guidance, lowering the top end of its revenue range due to potential upside being less likely. There are concerns about the impact of potential multisource oxybate generics entering the market by the end of 2025. Q & A Highlights Q: What's driving the strength of Xywav in idiopathic hypersomnia (IH), and is the growth sustainable? A: Renee Gala, President and COO, explained that the growth in Xywav is driven by strong execution across field teams and investments in consumer-targeted digital and media campaigns. These efforts have increased disease awareness and patient education, particularly in IH, where Xywav is the only FDA-approved therapy. The field nurse educator program also supports patients in titrating to an efficacious dose, enhancing persistence and overall growth. Q: Can you provide insights into the upcoming Phase 3 trial results for zanidatamab in gastroesophageal adenocarcinoma (GEA)? A: Bruce Cozadd, CEO, stated that Jazz typically provides top-line results indicating whether primary endpoints were met, while preserving detailed data for publication. Robert Iannone, EVP and Chief Medical Officer, added that recent experiences, like the IMforte trial, included commentary on endpoints and clinical meaningfulness, suggesting a similar approach for zanidatamab. Q: How do you view the competitive landscape for Zepzelca in second-line small cell lung cancer, and when do you expect stabilization? A: Renee Gala noted increased competition in the second-line setting and adoption of immunotherapy in first-line treatment, which delays progression to second-line. However, the potential approval of Zepzelca for first-line maintenance, based on IMforte study data, is expected to be practice-changing, offering a larger patient group and longer therapy duration. Q: What are your expectations for Epidiolex reaching blockbuster status, and are there any potential headwinds? A: Renee Gala expressed confidence in Epidiolex reaching blockbuster status, noting strong underlying demand despite typical inventory seasonality affecting growth comparisons. Philip Johnson, CFO, highlighted that less than 3% growth is needed to surpass $1 billion in revenue, with robust volume growth supporting this target. Q: What is the outlook for dordaviprone's approval and market potential? A: Bruce Cozadd mentioned ongoing conversations with the FDA, with a PDUFA target action date of August 18. Renee Gala emphasized excitement about the potential approval, noting that dordaviprone will primarily be administered in academic settings, requiring a focused launch team. The product has patent protection into the late 2030s, indicating a durable market opportunity. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
30-07-2025
- Business
- Yahoo
Morgan Stanley Reduces PT on Jazz Pharmaceuticals plc (JAZZ) from $166 to $165; Maintains ‘Buy' Rating
Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is included in our list of the 10 Most Undervalued Foreign Stocks to Buy According to Analysts. A closeup of pills in a pharmacy, representing the high quality medications of the company. Despite early-year challenges, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) remains a 'Buy'. On July 22, 2025, Morgan Stanley maintained its 'Buy' rating on JAZZ. However, the analyst lowered its price target slightly from $166 to $165. The analyst attributes its bullish stance to its expectation that the company will bounce back in the second half of the year, following a 10% miss in its Q1 net product sales. This expectation is driven by an extra shipping week in the Oncology segment, stronger demand for Rylaze due to pediatric protocol shifts, and seasonal strength in sleep disorder medication. Meanwhile, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) is expected to experience major clinical developments with HERIZON-GEA-01 data and the anticipated dordaviprone FDA decision. Furthermore, the upcoming leadership change boosts optimism. Looking ahead, the analyst's oncology revenue estimate exceeds the street's estimates. On the other hand, Wells Fargo reinforced this outlook, maintaining a 'Buy' rating on July 18 with a price target of $170. Operating in the United States, Europe, and other global markets, Jazz Pharmaceuticals plc (NASDAQ:JAZZ) develops and commercializes pharmaceutical products. It remains on our list of the most undervalued stocks. While we acknowledge the potential of JAZZ as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 14 Cheap Transportation Stocks to Buy According to Analysts and 10 Cheap Lithium Stocks to Buy According to Hedge Funds. Disclosure: None.
