Written By: Pharmacally Medical News Desk
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency recommended five new medicines for approval at its March 2026 meeting (23-26 March), spanning oncology, rare diseases, and paediatric care. These decisions highlight a continued focus on unmet medical needs, innovative therapies, and expanded treatment options, including orphan medicinal product designations for several agents.
Adstiladrin (nadofaragene firadenovec)
Marketing Authorisation Holder (MAH): Ferring Pharmaceuticals A/S
Indication: Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (CIS), with or without papillary tumours.
Clinical trial basis:
- Trial: Phase 3 Study (CS-003).
- Endpoint: Complete response rate in CIS patients, achieving clinically meaningful and durable responses.
Clinical value:Offers a gene therapy-based intravesical solution for BCG-failure patients with limited alternatives.
Imdylltra (tarlatamab): Orphan medicinal product
- MAH: Amgen Europe B.V.
- Indication: Relapsed extensive-stage small cell lung cancer (SCLC) after platinum-based chemotherapy
- Clinical trial basis:
- Trial: DeLLphi-301 (Phase 2)
- Endpoint: ORR and DoR achieved, with durable responses and emerging overall survival benefit
- Clinical value: First bispecific T-cell engager in SCLC, addressing a high unmet need in relapsed disease
Joenja (leniolisib) – Orphan Designation
MAH: Pharming Technologies B.V.
Indication: Activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents aged ≥12 years weighing ≥45 kg.
Clinical trial basis:
- Trial: Phase 3 APDS Study.
- Endpoint: Significant reduction in lymph node size, improved immunological markers, and reduced infections.
Clinical value:Directly targets the PI3Kδ hyperactivation underlying this rare primary immunodeficiency.
Zepzelca (lurbinectedin)-Orphan Designation
MAH: Pharma Mar S.A.
Indication: Maintenance treatment in extensive-stage small cell lung cancer after induction chemotherapy.
Clinical trial basis:
- Trial: Phase 3 ATLANTIS Study.
- Endpoint: Progression-free survival (PFS) benefit; overall survival (OS) primary endpoint not met, but secondary outcomes showed disease control.
Clinical value:Provides a novel maintenance option in relapsed/refractory SCLC with poor prognosis.
Bopediat (furosemide) – Paediatric Use Marketing Authorisation (PUMA)
MAH: Proveca Pharma Limited.
Indication: Oedema of cardiac, renal (including chronic kidney disease), or hepatic origin; hypertension; and situations requiring diuresis in paediatric patients.
Clinical trial basis:
- Basis: Hybrid application referencing originator data, supported by paediatric pharmacokinetic, safety, and efficacy studies.
- Endpoint: Confirmed appropriate exposure, safety profile, and efficacy with tailored dosing.
Clinical value:Enables age-appropriate orodispersible furosemide use in children, filling a gap in diuretic therapy.
The CHMP’s March 2026 recommendations balance groundbreaking innovations with practical adaptations, targeting cancers, rare immunodeficiencies, and paediatric needs. If approved by the European Commission, these will enhance the EU therapeutic landscape, improving patient access and outcomes.
Reference
Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 March 2026, 27 March 2026, https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-23-26-march-2026