Shots:
- The European Medicines Agency advanced multiple new drug approvals and late-stage recommendations in March 2026, spanning oncology, immunology, rare diseases, and women’s health, with key decisions involving Pharming, Immedica, Amgen, Gedeon Richter, UCB, and Sanofi
- The Committee for Medicinal Products for Human Use issued positive opinions for innovative therapies, including leniolisib (Joenja) for APDS, lurbinectedin + atezolizumab and tarlatamab for extensive-stage small cell lung cancer, supported by strong Phase II/III clinical evidence and long-term extension data demonstrating meaningful improvements in survival, immune function, and disease control
- The European Commission granted approvals to therapies such as estetrol-based Fylrevy for hormone replacement therapy, Kygevvi for thymidine kinase 2 deficiency, and Rezurock for chronic graft-versus-host disease, backed by clinical trials and real-world data showing improved symptom control, functional outcomes, motor milestone recovery, and durable response rates with favourable safety profiles across diverse patient populations in the EEA
Company: Pharming
Product: Joenja
Active Ingredient: Leniolisib
Disease: Activated Phosphoinositide 3-Kinase Delta Syndrome
Date: Mar 26, 2026
Shots:
- The CHMP has recommended Joenja for the treatment of pts (≥12yrs.) with APDS syndrome, with EC decision expected in Q2’26, which, if positive, would be valid across all 30 EEA member states
- Opinion was based on P-II/III trial of Joenja vs PBO in 31 APDS pts (≥12 years), showing significant improvement in immune dysregulation & immunodeficiency, plus was also supported by long-term OLE data in 37 pts treated with Joenja for a median of 3yrs.
- Leniolisib (PO), a PI3Kẟ inhibitor, is under regulatory review in Canada & other countries for APDS treatment & is also being investigated in 2 P-II trials for primary immunodeficiencies with immune dysregulation
Company: Immedica
Product: Zepzelca + Tecentriq
Active Ingredient: Lurbinectedin + Atezolizumab
Disease: ES-SCLC
Date: Mar 26, 2026
Shots:
- The CHMP has recommended Zepzelca + Tecentriq as a 1L therapy for adults with ES-SCLC whose disease remains stable after initial treatment with Tecentriq, carboplatin, & etoposide
- Opinion was based on P-III (IMforte) trial results, showing Zepzelca + Tecentriq reduced risk of progression or death by 46% & death risk by 27% vs Tecentriq alone
- Trial showed mOS of 13.2 vs 10.6mos. & mPFS of 5.4 vs 2.1 mos. for Zepzelca + Tecentriq vs Tecentriq alone, following four induction therapy cycles from randomization; data was presented at ASCO’25 & published in The Lancet
Company: Amgen
Product: Imdylltra
Active Ingredient: Tarlatamab
Disease: ES-SCLC
Date: Mar 26, 2026
Shots:
- The CHMP has recommended Imdylltra (tarlatamab) as a monotx. to treat adults with extensive-stage small cell lung cancer (ES-SCLC) whose disease relapsed during or after an initial treatment with Pt-based CT
- Opinion was based on the P-III study in 509 ES-SCLC pts post Pt therapy, where Imdylltra showed improved mOS (13.6 vs 8.3 mos.; ~40% lower death risk) and higher mPFS (4.2 vs 3.2 mos.) vs SoC
- Imdylltra is a bispecific T-cell engager that binds DLL3 on tumour cells and CD3 on T cells, activating immune response and driving tumour cell death via cytokine release and cytotoxic activity
Company: Gedeon Richter
Product: Fylrevy
Active Ingredient: Estetrol/ Donesta
Disease: Oestrogen Deficiency in Postmenopausal Women
Date: Mar 27, 2026
Shots:
- The EC has approved Fylrevy (estetrol/Donesta) as HRT for oestrogen deficiency symptoms in postmenopausal women, both non-hysterectomised (14.2 mg and 18.9 mg) and hysterectomized (18.9 mg) across EEA markets
- Fylrevy is an oral estetrol-based oestrogen therapy designed to treat a broad range of oestrogen deficiency symptoms, including VMS, in postmenopausal women
- Preclinical and clinical data show that estetrol (E4) has tissue-specific activity with minimal effects on the liver, hemostasis balance, and breast cell proliferation; its efficacy & safety in reducing VMS were confirmed in two pivotal P-III trials (E4COMFORT I & II) in 2,576 postmenopausal women across multiple regions. E4 is now expanding into menopause therapy
Company: UCB
Product: Kygevvi
Active Ingredient: Doxecitine and Doxribtimine
Disease: Thymidine Kinase 2 Deficiency
Date: Mar 26, 2026
Shots:
- The EC has approved Kygevvi (doxecitine and doxribtimine) under exceptional circumstances for adults & pediatric pts with TK2d whose symptom onset occurred ≤12yrs. of age
- Supportive evidence for approval came from pooled data from 2 studies of Kygevvi in pts with genetically confirmed TK2d & symptom onset ≤12yrs. The studies showed improved functional outcomes & survival vs matched external controls after long-term treatment (>5yrs.)
- Post-treatment, 84% (26/31) of patients regained ≥1 motor milestone. Ventilatory support initiation decreased (24% post vs 46% pre), with 22% discontinuing support. Feeding support also improved, with only 14% initiating post-treatment and some later discontinuing
Note:
- The following drugs have been granted CHMP; however, no PR was available:
- Adstiladrin (nadofaragene firadenovec)
- The following drugs have been approved; however, no PR was available:
- Ilumira (lutetium (177Lu) chloride)
Related Post: EMA Marketing Authorization of New Drugs in February 2026
