Lurbinectedin

On Friday, the EMA's drug advisory panel issued its latest raft of recommendations and regulatory updates, unveiling positive opinions for five new medicines.Heading the list was a recommendation to approve Amgen's Imdylltra (tarlatamab) to treat adults with extensive-stage small-cell lung cancer (ES-SCLC) whose disease relapsed during or after an initial treatment with platinum-based chemotherapy.The T-cell engager, which targets both DLL3 and CD3, was authorised in the same indication in the US in 2024, where it is marketed as Imdelltra. While the FDA's accelerated approval was based on the Phase II DeLLphi-301 study, the EMA's decision was supported by findings from the Phase III DeLLphi-304 trial. Data reviewed by the agency's Committee for Medicinal Products for Human Use (CHMP) showed that Imdylltra was associated with a significant improvement in median overall survival of 13.6 months, versus 8.3 months for standard care, corresponding to a 40% reduction in the risk of death. Meanwhile, median progression-free survival in the two groups was 4.2 months and 3.2 months, respectively.Similar to the US — where Amgen's drug carries a boxed warning for cytokine release syndrome (CRS) and neurologic toxicities — the EMA said that the product information for Amgen's drug will describe these risks and guidance on their management. Staying in ES-SCLC, the CHMP also recommended approval of PharmaMar's Zepzelca (lurbinectedin) for the maintenance treatment of patients whose disease has not progressed after first-line induction therapy. The alkylating agent, which was recently cleared for this use in the US, is indicated in combination with Roche's anti–PD-L1 Tecentriq (atezolizumab).Thumbs up for Sanofi's SC SarclisaAlso earning itself a CHMP nod was Sanofi and its on-body injector subcutaneous (SC) formulation of Sarclisa (isatuximab). The recommendation covers all four of the multiple myeloma indications currently held by the intravenous (IV) version of the anti-CD38 mAb in the EU.The automated hands-free device uses a 30-gauge hidden and retractable needle that is thinner than commonly used SC injection needles.At the American Society of Clinical Oncology (ASCO) meeting last year, Sanofi shared data from the Phase III IRAKLIA study showing that injectable Sarclisa achieved a 71.1% objective response rate after a median follow-up of 12 months, compared to 70.5% for the IV formulation, meeting the non-inferiority primary endpoint. Progression-free survival rates at 12 months were similar between the two formulations at 66.1% and 65.1%, respectively.Decisions go the way of Ferring, PharmingIn other decisions, the CHMP recommended conditional approval of Ferring Pharmaceuticals' gene therapy Adstiladrin (nadofaragene firadenovec) to treat adults with Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer with carcinoma in situ with or without papillary tumours.Adstiladrin has been on the market in the US for several years after winning FDA approval at the end of 2022.The CHMP also backed authorisation of Pharming's Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older weighing 45 kg or more. While the oral, selective PI3Kδ inhibitor is approved for this patient group in the US, efforts to expand use into children aged four to 11 years with the rare and progressive primary immunodeficiency were recently rejected by the FDA. Although Pharming is hopeful of addressing the US regulator's issues — which relate to clinical pharmacology and batch testing methodology — the paediatric approval had been expected to drive a Joenja growth inflection this year.Elsewhere, the CHMP issued a positive opinion recommending paediatric use of Proveca Pharma's Bopediat (furosemide) to treat oedema of cardiac or renal origin, oedema of hepatic origin and hypertension in children from birth to under 18 years of age with chronic kidney disease. Bopediat is a hybrid medicine of Lasilix Faible (furosemide), which has been authorised in France since 1977. The EMA noted that Proveca's drug contains the same active substance as Lasilix Faible, but it is available as orodispersible tablets and at a lower strength so that it can be given to young children.Re-examinations end badlyThe EMA on Friday also confirmed the recent decision by Anavex Life Sciences to withdraw an application for its Alzheimer's drug candidate blarcamesine. Having recommended refusing approval at the end of last year, the CHMP was in the process of re-examining the application — as requested by the drugmaker — when the company opted to pull the filing.Another re-examination resulting in a negative outcome went to Hetlioz (tasimelteon) and its developer Vanda Pharmaceuticals, who was seeking to expand use into people with a rare hereditary disorder, Smith-Magenis syndrome. The melatonin‑receptor agonist is already approved in the EU to treat totally blind adults with non-24-hour sleep-wake disorder.Having initially rejected use of Hetlioz in Smith-Magenis syndrome last November, the CHMP reaffirmed its decision at an extraordinary meeting on March 16. The committee expressed concerns about the study used to support the application, including the statistical analysis of the results and the way the trial had been conducted, which led to uncertainties about the drug's effects.Tecovirimat use restrictedThe EMA also said Friday that SIGA Technologies' antiviral tecovirimat should no longer be used for mpox. The drug, also known as TPOXX, was approved in the EU in 2022 to treat smallpox, mpox and cowpox based on data from animal studies.Subsequent trials conducted in humans — including the STOMP study sponsored by the US National Institutes of Health (NIH) — showed that tecovirimat did not heal lesions faster compared with placebo in people presenting with active mpox lesions, while it also failed to improve other outcomes, such as relieving pain or helping to clear the virus from the body faster.

Stockholm, Sweden, March 27, 2026 – Immedica today announces that Zepzelca® (lurbinectedin), in combination with atezolizumab, has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) as first-line treatment for adult patients with extensive-stage small cell lung cancer (ES-SCLC) whose disease has not progressed following induction therapy with atezolizumab, carboplatin and etoposide. Extensive-stage small cell lung cancer is an aggressive and fast-growing disease with a poor prognosis and limited treatment options, with the majority of patients diagnosed at an advanced stage. “Small cell lung cancer remains a highly challenging disease with significant unmet medical need,” said Anders Edvell, CEO of Immedica. “The CHMP’s positive opinion represents an important step towards bringing this new treatment option to patients in Europe.” The decision is based on results from the Phase 3 IMforte trial, which showed that the lurbinectedin and atezolizumab combination reduced the risk of disease progression or death by 46% and the risk of death by 27%, compared to atezolizumab maintenance therapy alone. Following four cycles of induction therapy, from the point of randomization the median overall survival (OS) for the combination regimen was 13.2 months versus 10.6 months (stratified hazard ratio [HR]=0.73; 95% CI: 0.57–0.95; p=0.0174). From the point of randomization, median progression-free survival (PFS) by independent assessment was 5.4 months versus 2.1 months, respectively (stratified HR=0.54, 95% CI: 0.43–0.67; p<0.0001). Safety was consistent with the known safety profiles of both treatments. The results were presented at the 2025 American Society of Clinical Oncology (ASCO) annual meeting and simultaneously published in The Lancet. The CHMP positive opinion will now be reviewed by the European Commission, which is responsible for the final decision on the marketing authorization in the EU. Immedica has a strategic partnership with PharmaMar, where Immedica holds the distribution rights to Zepzelca in the Nordics, the UK & Ireland, Central Eastern Europe as well as in the Middle East and North Africa. About small cell lung cancer (SCLC) SCLC is an aggressive form of lung cancer characterized by rapid growth, early metastasis, and limited therapeutic options. It accounts for about 10–15 % of all lung cancer cases and tends to present at an advanced stage, often requiring systemic therapy. Because of its aggressive nature and propensity for relapse the prognosis remains poor. About Zepzelca® (lurbinectedin) Zepzelca® (lurbinectedin), also known as PM1183, is an analog of the marine compound ET-736 isolated from the sea squirt Ecteinascidia turbinata in which a hydrogen atom has been replaced by a methoxy group. It is a selective inhibitor of the oncogenic transcription programs on which many tumors are particularly dependent. Together with its effect on cancer cells, lurbinectedin inhibits oncogenic transcription in tumor-associated macrophages, downregulating the production of cytokines that are essential for the growth of the tumor. Transcriptional addiction is an acknowledged target in those diseases, many of them lacking other actionable targets. About PharmaMar PharmaMar is a biopharmaceutical company headquartered in Madrid, Spain, specializing in the discovery, development, and commercialization of anticancer therapies derived from marine biodiversity. The company focuses its R&D efforts on rare and difficult-to-treat cancers, leveraging a proprietary platform of marine-derived compounds and strong collaborations with academic and industry partners. For more information, visit www.pharmamar.com About Immedica Immedica is a pharmaceutical company, headquartered in Stockholm, Sweden, focused on the commercialization of medicines for rare diseases and specialty care products. Immedica’s capabilities cover marketing and sales, compliance, pharmacovigilance, quality assurance, regulatory, medical affairs and market access, as well as a global distribution network serving patients in more than 50 countries. Immedica is fully dedicated to helping those living with diseases which have a large unmet medical need. Immedica’s therapeutic areas are within RARE metabolic, RARE hematology & oncology, RARE neurology, RARE Endocrinology and specialty care. Immedica was founded in 2018 and employs today around 180 people across Europe, the Middle East and the United States. Immedica is backed by the investment firms KKR and Impilo. For more information visit www.immedica.com. Immedica contact: Linda Holmström Head of HR & Communication linda.holmstrom@immedica.com We believe we have an important role to fill in society – bringing new, innovative treatments to patients with rare diseases. Focus areas are within RARE metabolic, RARE neurology, RARE hematology & oncology and specialty care. We operate products from phase 2 and all the way to mature products. ESG is an integrated part of our business model. Stay up to date with our latest news. Telephone: +46 (0) 8 533 39 500 E-mail: info@immedica.com