Topotecan Hydrochloride

Final data analysis from Phase 3 MIRASOL trial after 30.5-month median follow-up demonstrated 32% reduction in risk of death with ELAHERE compared to chemotherapy in folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) Data presented in a late-breaking oral presentation at the Society of Gynecologic Oncology (SGO) Annual Meeting on Women's Cancer in Seattle NORTH CHICAGO, Ill., March 15, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the final analysis of the confirmatory Phase 3 MIRASOL trial evaluating the efficacy and safety of ELAHERE® (mirvetuximab soravtansine-gynx) in women with folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer (PROC) compared to chemotherapy. At 30.5 months median follow-up, treatment with ELAHERE continued to show significant improvements in progression-free survival (PFS) and overall survival (OS) compared to investigator's choice (IC) chemotherapy.1 Ovarian cancer patients often present with late-stage disease and are historically first treated with platinum-based chemotherapy, which they may become resistant to and require another therapy, such as ELAHERE.2 "Ovarian cancer can be devastating, and when cancer cells stop responding to chemotherapy patients may feel hopeless about their journey. The data presented today reinforce the importance of ELAHERE as a transformative therapy for patients with limited options," said Svetlana Kobina, MD, PhD, vice president, oncology medical affairs, AbbVie. "We remain steadfast in our commitment to bring forward innovative therapies that improve the lives of patients with difficult-to-treat cancers." In the United States, ovarian cancer is the leading cause of death from gynecological cancers.3 Each year, approximately 20,000 women are diagnosed.4 Unfortunately, most patients develop platinum-resistant disease, which is difficult to treat.5 In this setting, single-agent chemotherapies are associated with minimal survival benefit while adding significant toxicity burden.6 The Phase 3 MIRASOL study included 453 patients with high-grade serous epithelial PROC whose tumors express high levels of FRα and had been treated with up to three prior therapies.1 Key findings from the 30.5-month median follow-up include: ELAHERE treatment achieved superior efficacy versus IC chemotherapy, with a median PFS of 5.59 months versus 3.98 months, representing a 37% reduction in the risk of tumor progression or death (HR 0.63; [95% CI: 0.51, 0.79]) and a higher objective response rate of 41.9% versus 15.9%. Superior and clinically meaningful overall survival for patients receiving ELAHERE (median 16.85 months) compared to IC chemotherapy (median 13.34 months), representing a 32% reduction in the risk of death (HR 0.68 [95% CI: 0.54, 0.84]). Other endpoints included safety and duration of response (DOR), which were consistent with the primary data analysis at 13.1-months median follow-up. The most common treatment-emergent adverse events (TEAEs) occurring in at least 20% of patients in the ELAHERE arm were blurred vision, keratopathy, abdominal pain, fatigue, diarrhea, dry eye, constipation, nausea and peripheral neuropathy. Compared with IC chemotherapy, treatment with ELAHERE was overall associated with lower rates of grade ≥3 TEAEs, serious AEs and discontinuations due to AEs. "The final data showcase the significant improvement in overall survival benefit of treatment with ELAHERE compared to standard of care chemotherapy," said investigator and presenter, Toon Van Gorp, MD, PhD, Professor of Gynecologic Oncology, University of Leuven. "The significant improvements in survival, along with the well-characterized safety profile, reinforce ELAHERE as an emerging standard of care for difficult-to-treat ovarian cancer and warrants further study of this medicine in earlier treatment settings." A separate analysis from the Phase 3 MIRASOL study evaluating the impact of [ELAHERE] treatment-emergent ocular events on patient-reported health-related quality of life (HRQoL), will be shared during an oral presentation March 17 at the SGO Annual Meeting scientific plenary session. ELAHERE was granted full approval by the U.S. Food and Drug Administration in March 2024 and was approved by the European Commission in November 2024. Marketing Authorization Applications for ELAHERE are also under review in multiple other countries. About the Phase 3 MIRASOL Trial MIRASOL is a randomized Phase 3 trial of ELAHERE versus investigator's choice (IC) of single-agent chemotherapy (weekly paclitaxel, pegylated liposomal doxorubicin, or topotecan). Eligibility criteria include patients with PROC whose tumors express high levels of FRα, using the Ventana FOLR1 RxDx Assay, and who have been treated with up to three prior regimens. The primary endpoint of this trial is progression-free survival (PFS) by investigator assessment. Key secondary endpoints include objective response rate (ORR) and overall survival (OS). The trial enrolled 453 patients. Patients were stratified by number of prior lines of therapy (14% had one prior line of therapy, 39% had two prior lines of therapy, and 47% had three prior lines of therapy) and by IC chemotherapy, with paclitaxel as the most commonly chosen (41%), followed by PLD (36%) and topotecan (23%). Sixty-two percent of patients received prior bevacizumab; 55% received a prior PARP inhibitor. More information can be found on (NCT04209855). About ELAHERE ELAHERE (mirvetuximab soravtansine-gynx) is a first-in-class antibody-drug conjugate (ADC) comprising a folate receptor alpha-binding antibody, cleavable linker, and the maytansinoid payload DM4, a potent tubulin inhibitor designed to kill the targeted cancer cells. Patients requiring access support may call 1-833-ELAHERE or visit . ELAHERE U.S. USE and IMPORTANT SAFETY INFORMATION7 What is ELAHERE? ELAHERE is a prescription medicine used to treat adults with folate receptor-alpha positive ovarian cancer, fallopian tube cancer, or primary peritoneal cancer who: have not responded to or are no longer responding to treatment with platinum-based chemotherapy and have received 1 to 3 prior types of chemotherapy. Your healthcare provider will perform a test to make sure that ELAHERE is right for you. It is not known if ELAHERE is safe and effective in children. IMPORTANT SAFETY INFORMATION What is the most important information I should know about ELAHERE? ELAHERE can cause serious side effects, including: Eye problems. Eye problems are common with ELAHERE and can also be severe. Tell your healthcare provider right away if you develop any eye problems during treatment with ELAHERE, including blurred vision, dry eyes, sensitivity to light, eye pain, eye redness, or new or worsening vision changes. Your healthcare provider will send you to see an eye care professional to check your eyes before you start treatment with ELAHERE, during treatment with ELAHERE, and as needed for any worsening signs and symptoms of eye problems. Your healthcare provider will prescribe steroid eye drops and lubricating eye drops before you start and during your treatment with ELAHERE. You should use eye drops as directed by your healthcare provider. Do not wear contact lenses throughout your treatment with ELAHERE unless you are told to use them by your healthcare provider. What should I tell my healthcare provider before receiving ELAHERE? Tell your healthcare provider about all of your medical conditions, including if you: have vision or eye problems. have numbness or tingling in your hands or feet. have liver problems. are pregnant or plan to become pregnant. ELAHERE can harm your unborn baby. Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with ELAHERE. Patients who are able to become pregnant: Your healthcare provider should do a pregnancy test before you start treatment with ELAHERE. You should use an effective birth control (contraception) during treatment and for 7 months after your last dose of ELAHERE. are breastfeeding or plan to breastfeed. It is not known if ELAHERE passes into your breast milk. Do not breastfeed during treatment and for 1 month after your last dose of ELAHERE. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking certain other medicines during treatment with ELAHERE may cause side effects. What are the possible side effects of ELAHERE? ELAHERE can cause serious side effects, including: Eye problems. Eye problems are common with ELAHERE and can also be severe. Tell your healthcare provider right away if you develop any eye problems during treatment with ELAHERE, including blurred vision, dry eyes, sensitivity to light, eye pain, eye redness, or new or worsening vision changes. Lung problems (pneumonitis). ELAHERE can cause severe or life-threatening inflammation of the lungs that may lead to death. Tell your healthcare provider right away if you get new or worsening symptoms, including trouble breathing, shortness of breath, cough, or chest pain. Peripheral neuropathy. Nerve problems called peripheral neuropathy are common during treatment with ELAHERE and can also be severe. Your healthcare provider will monitor you for signs and symptoms of nerve problems. Tell your healthcare provider if you get new or worsening numbness, tingling, burning sensation or pain in your hands or feet or muscle weakness. The most common side effects and abnormal labs of ELAHERE include: Your healthcare provider may change your dose of ELAHERE, delay treatment, or completely stop treatment if you have certain side effects. These are not all of the possible side effects of ELAHERE. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit or call 1‑800‑FDA‑1088. Please see Full Prescribing Information, including Boxed WARNING and Medication Guide. About AbbVie in Oncology At AbbVie, we are committed to transforming standards of care for patients living with difficult-to-treat cancers. We are advancing a dynamic pipeline of investigational therapies across a range of cancer types in both blood cancers and solid tumors. We are focusing on creating targeted medicines that either impede the reproduction of cancer cells or enable their elimination. We achieve this through various, targeted treatment modalities and biology interventions, including small molecule therapeutics, antibody-drug conjugates (ADCs), Immuno-Oncology-based therapeutics, multi-specific antibody and in situ CAR-T platforms. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines. Today, our expansive oncology portfolio comprises approved and investigational treatments for a wide range of blood and solid tumors. We are evaluating more than 20 investigational medicines in multiple clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit . About AbbVie AbbVie's mission is to discover and deliver innovative medicines and solutions that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas including immunology, oncology, neuroscience and eye care – and products and services in our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at . Follow @abbvie on LinkedIn, Facebook, Instagram, X (formerly Twitter), and YouTube. AbbVie Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2024 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. Disclosure: Dr. Van Gorp has financial interests related to AbbVie. References: Van Gorp T, et al. Final overall survival analysis among patients with FRα-positive platinum-resistant ovarian cancer (PROC) treated with mirvetuximab soravtansine (MIRV) vs. investigator's choice chemotherapy (ICC) in the Phase 3 MIRASOL (GOG 3045/ENGOT-ov55) study. Data presented at the 2025 Society of Gynecologic Oncology Annual Meeting on Women's Cancer. Hanker, LC, et al. The impact of second to sixth line therapy on survival of relapsed ovarian cancer after primary taxane/platinum-based therapy, Annals of Oncology, Volume 23, Issue 10, 2012. WHO International Agency for Research on Cancer. GLOBOCAN 2022. Cancer Today. Absolute numbers, Mortality, Females, age [0-74], in 2022. Continents. Available here. Whittemore AS. Characteristics relating to ovarian cancer risk: implications for prevention and detection. Gynecol Oncol. 1994 Dec;55(3 Pt 2):S15-9. doi: 10.1006/gyno.1994.1334. PMID: 7835800. Hanker, LC, et al. The impact of second to sixth line therapy on survival of relapsed ovarian cancer after primary taxane/platinum-based therapy, Annals of Oncology, Volume 23, Issue 10, 2012. Matulonis, UA, er al. Efficacy and safety of mirvetuximab soravtansine in patients with platinum-resistant ovarian cancer with high folate receptor alpha expression: Results from the SORAYA Study. J Clin Oncol. 2023 May 1;41(13):2436-2445. doi: 10.1200/JCO.22.01900. Epub 2023 Jan 30. PMID: 36716407; PMCID: PMC10150846. ELAHERE [package insert]. North Chicago, IL: AbbVie Inc: 2025. SOURCE AbbVie WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphelia said it will continue to make Kizfizo available to paediatric patients under certain programmes and clinical trials. Credit: Andrii Yalanskyi via Getty Images. Orphelia Pharma is exploring alternative regulatory pathways for Kizfizo (temozolomide) following a negative opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). The latest decision follows another negative opinion for Orphelia’s marketing authorisation application (MAA) in November 2024. The CHMP determined that Kizfizo failed to demonstrate sufficient clinical benefit to establish a positive benefit-risk balance. The agency cited modest response rates and the lack of convincing evidence that Kizfizo offered significant advantages over existing therapies for relapsed/refractory neuroblastoma. In response to the initial negative opinion, Orphelia requested a re-examination of the decision in December 2024. However, after a re-evaluation on 27 February 2025, the CHMP upheld its initial stance, stating that the committee’s concerns were not resolved. The company shared this update in a press release today (12 March). Orphelia emphasised that it will continue to make Kizfizo available to paediatric patients under compassionate use or early access programmes as well as to patients participating in ongoing clinical trials. “Considering the important unmet medical need, we are currently exploring all available options to make Kizfizo available to the paediatric patients,” said Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma in the 12 March announcement. Neuroblastoma is a type of cancer that forms in certain types of nerve tissue, mostly affecting children under the age of five. Neuroblastoma is the second most common solid tumour in children after brain tumours, according to the UK-based charity The Children & Young People’s Cancer Association (CCLG). Kizfizo was designed as a hybrid medicine similar to MSD’s chemotherapy agent Temodal but is formulated as a liquid for easier administration, as many babies and children cannot take oral tablets. Orphelia presented clinical data from studies enrolling 102 patients up to 21 years old. The Phase I TEMOkids (NCT04610736) and Ped-TMZ studies (NCT04467346) demonstrated safety, pharmacokinetics, and efficacy. The ongoing Phase I/II BEACON2 study is evaluating Kizfizo in combination with chemotherapies irinotecan or topotecan. Originally indicated exclusively for the treatment of glioblastoma in adults in the US, temozolomide has been used off label to treat neuroblastoma patients for years. The drug has been approved for treating glioblastoma in adults and children in Europe since 2010. Kizfizo was granted early access authorisation by the French authorities in March 2022 and has received orphan drug designation from the EMA and the US Food and Drug Administration (FDA). The only approved drug in Orphelia’s portfolio is Kigabeq (vigabatrin), its paediatric formulation of vigabatrin, which has an EU authorisation as a monotherapy for West syndrome and for use as a combination treatment for resistant partial epilepsy.