Shots:
- Innovation in drug development continues to redefine the future of healthcare, fueling bold scientific partnerships and transforming how diseases are understood, managed, and treated across every therapeutic frontier
- In November, the EMA issued positive CHMP opinions for six significant therapies and 2 EU approval: Sanofi’s Teizeild (Teplizumab) to delay the onset of stage 3 type 1 diabetes, and Ionis and Otsuka’s Dawnzera (Donidalorsen) for the routine prevention of HAE attacks
- In November 2025, the EMA authorised eight new therapies in the EU, compared with the five approvals issued by the US FDA that month. These decisions reflect sustained global momentum in making innovative and advanced treatments available to patients.
Company: Ionis and Otsuka
Product: Dawnzera
Active Ingredient: Donidalorsen
Disease: Hereditary Angioedema
Date: Nov 13, 2025
Shots:
- The CHMP has recommended Dawnzera for the routine prevention of HAE attacks in pts (≥12yrs.) following the US FDA approval in Aug 2025; EC’s decision is expected in Q1’26
- Opinion was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate even when Dawnzera is self-administered via an autoinjector
- Otsuka licensed Dawnzera from Ionis in Dec 2023 for exclusive commercialization in Europe & later, in June 2024, secured exclusive rights for the APAC region, incl. Japan
Company: Fondazione Telethon
Product: Waskyra
Active Ingredient: Etuvetidigene Autotemcel
Disease: Wiskott-Aldrich syndrome
Date: Nov 13, 2025
Shots:
- The CHMP has recommended Waskyra, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS)
- Waskyra is a single-administration therapy that uses a patient’s CD34+ hematopoietic stem and progenitor cells, modified with a lentiviral vector carrying the WAS gene
- The therapy will be available to pts at IRCCS Ospedale San Raffaele, where the clinical trial was conducted. The BLA for WAS remains under FDA review
Company: BioNet
Product: VacPertagen
Active Ingredient: Pertussis Vaccine (Recombinant, Acellular, Component, Adsorbed)
Disease: Pertussis Disease
Date: Nov 13, 2025
Shots:
- The CHMP has recommended VacPertagen as a booster aganist Pertussis for pts (age≥12) and for passive infant protection via maternal immunization during pregnancy
- VacPertagen is an acellular pertussis vaccine with two components, including recombinant Pertussis Toxin (PTgen) and filamentous haemagglutinin (FHA), providing targeted protection without unnecessary antigens
- VacPertagen’s benefits have been demonstrated in three clinical studies involving adults, adolescents, and pregnant women
Company: Sanofi
Product: Teizeild
Active Ingredient: Teplizumab
Disease: Type 1 Diabetes
Date: Nov 13, 2025
Shots:
- The CHMP has recommended Teizeild to delay the onset of stage 3 type 1 diabetes (T1D) in adult & pediatric pts (≥8yrs.) with stage 2 T1D; regulatory review is ongoing in the other regions
- Opinion was based on the P-II (TN-10) trial assessing Teizeild (QD, n=44) vs PBO (n=32) for 14 days in 76 T1D pts, where Teizeild delayed stage 3 onset by a median of ~2yrs. & kept 57% vs 28% of pts in stage 2
- Additionally, Sanofi is pausing its Teizeild application for recently diagnosed stage 3 T1D & evaluating next steps following CHMP’s positive opinion & discussions with the EMA
Company: Bayer
Product: Lynkuet
Active Ingredient: Elinzanetant
Disease: Vasomotor Symptoms
Date: Nov 17, 2025
Shots:
- The EC has approved Lynkuet for the treatment of mod. to sev. vasomotor symptoms due to menopause or caused by adj. endocrine therapy related to breast cancer; regulatory filings are ongoing in other regions
- Approval was based on P-III (OASIS) program assessing Lynkuet (120mg, QD, PO), which consists of OASIS-1 (n=396), OASIS-2 (n=400) & OASIS-3 (n=628) in postmenopausal women (40-65yrs.) as well as OASIS-4 (n=474) in women with VMS due to endocrine therapy
- Lynkuet met all 1 & 2EPs in all 4 studies & showed favorable safety, with OASIS 1 & 2 data published in The JAMA, OASIS-3 in The JAMA Internal Medicine, & OASIS-4 in The NEJM
Company: Insmed
Product: Brinsupri
Active Ingredient: Brensocatib
Disease: Non-Cystic Fibrosis Bronchiectasis
Date: Nov 18, 2025
Shots:
- The EC has approved Brinsupri (25mg) for treating NCFB pts (≥12yrs.), with ≥2 exacerbations in the prior 12mos. under EMA’saccelerated assessment pathway; Application is under PMDA & MHRA review, & Insmed is planning to work with EU authorities to enable patient access starting in early 2026
- Approval was based on extensive clinical data, incl. P-III (ASPEN) trial (n=1721) & P-II (WILLOW) trial (n=256) assessing Brinsupri (10 or 25mg, PO) vs PBO in NCFB pts for 52 & 24wks., respectively
- In ASPEN, Brinsupri (25mg) reduced annual exacerbations by 19.4%, showing benefits across exacerbation-related 2EPs with delayed first exacerbation & increased exacerbation-free pts; pts dosed with 25mg showed lower lung function decline per FEV₁ at wk. 52
Note: The following drug has been granted CHMP; however, no PR was available:
- Inluriyo (Imlunestrant)
- GalenVita (Germanium (68ge) Chloride / Gallium (68ga) Chloride)
Related Post: Insights+: EMA Marketing Authorization of New Drugs in October 2025
