Target Action (PDUFA) Date set for October 7, 2025
Application based on data from IMforte, the first Phase 3 trial demonstrating statistically significant and clinically meaningful improvements in both progression-free and overall survival in the ES-SCLC first-line maintenance setting
Jazz to host investor webcast on Tuesday, June 10 at 4:30 p.m. EDT / 9:30 p.m. IST to review Zepzelca data
For U.S. media and investors only
DUBLIN, Ireland I June 10, 2025 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the U.S. Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for Zepzelca® (lurbinectedin) in combination with atezolizumab (Tecentriq®) as a first-line maintenance treatment for people with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin, and etoposide for Priority Review, with a Prescription Drug User Fee Act (PDUFA) action date of October 7, 2025. Priority Review is granted to applications for drugs that have the potential to significantly improve the treatment, prevention, or diagnosis of serious conditions.
“The FDA’s Priority Review designation for Zepzelca in combination with atezolizumab as a first-line maintenance treatment highlights the urgent need for new approaches and the potential benefit of Zepzelca for patients with extensive-stage small cell lung cancer, a disease with limited therapeutic options and high unmet need,” said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. “We are pleased to have received this review designation after presenting the IMforte trial data at ASCO 2025 with simultaneous publication in The Lancet. Together, these milestones bring us a step closer to potentially offering patients a new first-line maintenance option that could help extend the time they live without their disease progressing.”
The sNDA submission is based on results from the Phase 3 IMforte trial of Zepzelca in combination with atezolizumab, which met both primary endpoints, demonstrating statistically significant improvements in progression-free survival (PFS) and overall survival (OS) compared to atezolizumab alone. Following induction therapy with carboplatin, etoposide and atezolizumab, patients who did not have disease progression were randomized to receive Zepzelca plus atezolizumab or atezolizumab alone. From the point of randomization, which occurred after patients completed the induction phase of four cycles of platinum-based chemotherapy, the median PFS was 5.4 months for the Zepzelca plus atezolizumab combination versus 2.1 months for atezolizumab alone (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001), and median OS was 13.2 months versus 10.6 months (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). The combination reduced the risk of disease progression or death by 46% and the risk of death by 27% compared to atezolizumab alone. The Zepzelca plus atezolizumab combination had no new or unexpected safety signals.
Webcast Details
IMforte trial results were first presented in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published simultaneously in The Lancet. The Company will host an investor webcast on June 10 at 4:30 p.m. EDT / 9:30 p.m. IST to review these data. The webcast will include commentary from a leading small cell lung cancer expert and Company senior management. The webcast may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com.
About Small Cell Lung Cancer
In the U.S., approximately 13 percent of lung cancers are small cell.1 Approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. each year.1,2 The risk for developing SCLC is much higher among current or former tobacco smokers; however, SCLC can also be caused by exposure to secondhand smoke, asbestos, some inhaled chemicals, radiation and air pollution. People with a family history of lung cancer may also be at a higher risk, too.1 SCLC is the most aggressive form of lung cancer and it tends to spread quickly to other parts of the body including the brain, liver and bone.2,3 A large percentage of SCLC patients on treatment briefly achieve a response, although the cancer often returns and is usually more aggressive and resistant to regimens that were previously effective.2
About Zepzelca® (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and potentially cell death.4
The FDA approved Zepzelca under accelerated approval in June 2020 for the treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. The approval is based on overall response rate (ORR) and duration of response (DOR) demonstrated in an open-label, monotherapy clinical study. In December 2021, Jazz and PharmaMar announced the initiation of LAGOON, a confirmatory Phase 3 clinical trial of Zepzelca for the treatment of patients with relapsed SCLC. If positive, LAGOON could confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following 1L treatment with a platinum-based regimen and support full approval in the U.S.
ZEPZELCA (lurbinectedin) for injection 4 mg, is indicated for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy.
This indication is approved under accelerated approval based on ORR and DOR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see accompanying full Prescribing Information.
ZEPZELCA is a trademark of Pharma Mar, S.A. used by Jazz Pharmaceuticals under license.
Tecentriq (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.
1 American Cancer Society. Small cell lung cancer causes, risk factors, and prevention. https://www.cancer.org/content/dam/CRC/PDF/Public/8709.00.pdf. Updated May 16, 2016. Accessed June 10, 2025.
2 American Cancer Society. What is lung cancer? https://www.cancer.org/cancer/lung-cancer/about/what-is.html. Updated October 1, 2019. Accessed June 10, 2025.
3 American Cancer Society. Small cell lung cancer stages. https://www.cancer.org/cancer/lung-cancer/detection-diagnosis-staging/staging-sclc.html. Updated October 1, 2019. Accessed June 10, 2025.
4 ZEPZELCA (lurbinectedin) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.
SOURCE: Jazz Pharmaceuticals
Post Views: 1,073
Target Action (PDUFA) Date set for October 7, 2025
Application based on data from IMforte, the first Phase 3 trial demonstrating statistically significant and clinically meaningful improvements in both progression-free and overall survival in the ES-SCLC first-line maintenance setting
Jazz to host investor webcast on Tuesday, June 10 at 4:30 p.m. EDT / 9:30 p.m. IST to review Zepzelca data
For U.S. media and investors only
DUBLIN, Ireland I June 10, 2025 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced the U.S. Food and Drug Administration (FDA) has accepted the supplemental New Drug Application (sNDA) for Zepzelca® (lurbinectedin) in combination with atezolizumab (Tecentriq®) as a first-line maintenance treatment for people with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin, and etoposide for Priority Review, with a Prescription Drug User Fee Act (PDUFA) action date of October 7, 2025. Priority Review is granted to applications for drugs that have the potential to significantly improve the treatment, prevention, or diagnosis of serious conditions.
“The FDA’s Priority Review designation for Zepzelca in combination with atezolizumab as a first-line maintenance treatment highlights the urgent need for new approaches and the potential benefit of Zepzelca for patients with extensive-stage small cell lung cancer, a disease with limited therapeutic options and high unmet need,” said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. “We are pleased to have received this review designation after presenting the IMforte trial data at ASCO 2025 with simultaneous publication in The Lancet. Together, these milestones bring us a step closer to potentially offering patients a new first-line maintenance option that could help extend the time they live without their disease progressing.”
The sNDA submission is based on results from the Phase 3 IMforte trial of Zepzelca in combination with atezolizumab, which met both primary endpoints, demonstrating statistically significant improvements in progression-free survival (PFS) and overall survival (OS) compared to atezolizumab alone. Following induction therapy with carboplatin, etoposide and atezolizumab, patients who did not have disease progression were randomized to receive Zepzelca plus atezolizumab or atezolizumab alone. From the point of randomization, which occurred after patients completed the induction phase of four cycles of platinum-based chemotherapy, the median PFS was 5.4 months for the Zepzelca plus atezolizumab combination versus 2.1 months for atezolizumab alone (stratified HR = 0.54, 95% CI: 0.43–0.67; p < 0.0001), and median OS was 13.2 months versus 10.6 months (stratified hazard ratio [HR] = 0.73; 95% CI: 0.57–0.95; p = 0.0174). The combination reduced the risk of disease progression or death by 46% and the risk of death by 27% compared to atezolizumab alone. The Zepzelca plus atezolizumab combination had no new or unexpected safety signals.
Webcast Details
IMforte trial results were first presented in an oral session at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting and published simultaneously in The Lancet. The Company will host an investor webcast on June 10 at 4:30 p.m. EDT / 9:30 p.m. IST to review these data. The webcast will include commentary from a leading small cell lung cancer expert and Company senior management. The webcast may be accessed from the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com.
About Small Cell Lung Cancer
In the U.S., approximately 13 percent of lung cancers are small cell.1 Approximately 30,000 new cases of small cell lung cancer (SCLC) are reported in the U.S. each year.1,2 The risk for developing SCLC is much higher among current or former tobacco smokers; however, SCLC can also be caused by exposure to secondhand smoke, asbestos, some inhaled chemicals, radiation and air pollution. People with a family history of lung cancer may also be at a higher risk, too.1 SCLC is the most aggressive form of lung cancer and it tends to spread quickly to other parts of the body including the brain, liver and bone.2,3 A large percentage of SCLC patients on treatment briefly achieve a response, although the cancer often returns and is usually more aggressive and resistant to regimens that were previously effective.2
About Zepzelca® (lurbinectedin)
Zepzelca is an alkylating drug that binds guanine residues within DNA. This triggers a cascade of events that can affect the activity of DNA binding proteins, including some transcription factors, and DNA repair pathways, resulting in disruption of the cell cycle and potentially cell death.4
The FDA approved Zepzelca under accelerated approval in June 2020 for the treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy. The approval is based on overall response rate (ORR) and duration of response (DOR) demonstrated in an open-label, monotherapy clinical study. In December 2021, Jazz and PharmaMar announced the initiation of LAGOON, a confirmatory Phase 3 clinical trial of Zepzelca for the treatment of patients with relapsed SCLC. If positive, LAGOON could confirm the benefit of Zepzelca in the treatment of SCLC when patients progress following 1L treatment with a platinum-based regimen and support full approval in the U.S.
ZEPZELCA (lurbinectedin) for injection 4 mg, is indicated for the treatment of adult patients with metastatic SCLC with disease progression on or after platinum-based chemotherapy.
This indication is approved under accelerated approval based on ORR and DOR. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see accompanying full Prescribing Information.
ZEPZELCA is a trademark of Pharma Mar, S.A. used by Jazz Pharmaceuticals under license.
Tecentriq (atezolizumab) is a registered trademark of Genentech, a member of the Roche Group.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit www.jazzpharmaceuticals.com for more information.
1 American Cancer Society. Small cell lung cancer causes, risk factors, and prevention. https://www.cancer.org/content/dam/CRC/PDF/Public/8709.00.pdf. Updated May 16, 2016. Accessed June 10, 2025.
2 American Cancer Society. What is lung cancer? https://www.cancer.org/cancer/lung-cancer/about/what-is.html. Updated October 1, 2019. Accessed June 10, 2025.
3 American Cancer Society. Small cell lung cancer stages. https://www.cancer.org/cancer/lung-cancer/detection-diagnosis-staging/staging-sclc.html. Updated October 1, 2019. Accessed June 10, 2025.
4 ZEPZELCA (lurbinectedin) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.
SOURCE: Jazz Pharmaceuticals
Post Views: 1,073
