Shots:
- The European Commission approved multiple therapies across vaccines, rare diseases, endocrinology, and immunology, including products from BioNet, Sanofi, Fondazione Telethon, IntraBio, and Ionis / Otsuka.
- The Committee for Medicinal Products for Human Use also issued positive opinions for late-stage assets from UCB and Gedeon Richter, with additional recommendations supporting Sanofi’s Rezurock.
- Decisions were backed by strong Phase II/III data showing delayed disease progression, reduced attack rates, improved survival, and favorable safety profiles across diverse patient populations in the EU
Company: BioNet
Product: VacPertagen
Active Ingredient: Pertussis Vaccine (Recombinant, Acellular, Component, Adsorbed)
Disease: Pertussis Disease
Date: Jan 09, 2026
Shots:
- The EC has approved VacPertagen as a booster aganist Pertussis for pts (age≥12) and for passive infant protection via maternal immunization during pregnancy
- CHMP recommendation was supported by data from three clinical trials and nine supportive studies, all consistently demonstrating that VacPertagen is safe for use in teenagers, adults, and pregnant women across populations
- VacPertagen is an acellular pertussis vaccine with two components, including recombinant Pertussis Toxin (PTgen) and filamentous haemagglutinin (FHA), providing targeted protection without unnecessary antigens
Company: Sanofi
Product: Teizeild
Active Ingredient: Teplizumab
Disease: Type 1 Diabetes
Date: Jan 12, 2026
Shots:
- The EC has approved Teizeild (Tzield outside the EU) 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
- Approval 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. vs PBO & kept 57% vs 28% of pts in stage 2
- Additionally, Sanofi is pausing its Teizeild application for recently diagnosed stage 3 T1D & is evaluating next steps with the EMA
Company: Fondazione Telethon
Product: Waskyra
Active Ingredient: Etuvetidigene Autotemcel
Disease: Wiskott-Aldrich Syndrome
Date: Jan 09, 2026
Shots:
- The EC has approved Waskyra (etuvetidigene autotemcel), 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 EC’s decision follows a CHMP opinion issued in November 2025 recommending marketing authorization, with the same therapy also approved by the U.S. FDA in December 2025; underscoring the effectiveness and global uniqueness of its research and development model
Company: IntraBio
Product: Aqneursa
Active Ingredient: Levacetylleucine
Disease: Niemann-Pick Disease Type C
Date: Jan 21, 2026
Shots:
- The EC has approved Aqneursa (levacetylleucine), available as 1g granules for oral suspension, with or without miglustat for treating adults & pediatric pts (≥6yrs.; ≥20kg) with NPC
- Approval was backed by P-III (IB1001-301) crossover study assessing Aqneursa vs PBO in NPC pts, which showed improved neurological symptoms & function after 12wks. as measured by SARA, with the ongoing OLE phase showing sustained disease-modifying & neuroprotective effects
- Also, observational analyses vs a natural history cohort showed Aqneursa reduced annual disease progression by 118% at 1yr. measured by the 5-domain NPC-CSS, with a comparable sustained effect observed after 2yrs.
Company: Ionis and Otsuka
Product: Dawnzera
Active Ingredient: Donidalorsen
Disease: Hereditary Angioedema
Date: Jan 21, 2026
Shots:
- The EC has approved Dawnzera (SC autoinjector; Q4W/Q8W) for the routine prevention of HAE attacks in pts (≥12yrs.)
- Approval was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate, with 94% overall mean monthly reduction at 1yr. in the OASISplus OLE study
- Approval triggered $15M milestone payment to Ionis, along with tiered royalties of up to 30% on product sale under its deal with Otsuka, that granted Otsuka exclusive rights to Dawnzera across EU & APAC
Company: Sanofi
Product: Rezurock
Active Ingredient: Belumosudil
Disease: Chronic Graft-vs-Host Disease
Date: Jan 29, 2026
Shots:
- The CHMP has recommended Rezurock (200mg) for conditional approval in chronic GVHD pts (≥12yrs., ≥40kg), with EC’s decision expected in the coming wks.
- Opinion was supported by extensive clinical data, incl. the P-II (ROCKstar) trial, which showed an improved ORR of 74% in chronic GVHD pts post–stem cell transplant & after ≥2 prior systemic therapies
- Under CHMP’s positive opinion, Sanofi will conduct a confirmatory randomized study & will continue evaluating Rezurock in additional age groups & indications, incl. pediatric chronic GVHD (≥1yr.) & chronic lung allograft dysfunction
Company: UCB
Product: Kygevvi
Active Ingredient: Doxecitine + Doxribtimine
Disease: Thymidine Kinase 2 Deficiency
Date: Jan 29, 2026
Shots:
- The CHMP has recommended Kygevvi (PO) under exceptional circumstances for adults & pediatric pts with TK2d whose symptom onset occurred ≤12yrs. of age, supported through EMA’s PRIME scheme. EC decision is expected by Q2’26
- Supportive evidence came from pooled data from 2 studies of Kygevvi in 39 pts with genetically confirmed TK2d & symptom onset ≤12yrs. The studies showed improved functional outcomes & survival vs matched external controls after long-term treatment (>5yrs.)
- Doxecitine & doxribtimine integrate into skeletal muscle mitochondrial DNA, restoring mtDNA copy number & improving muscle function in pts with TK2d
Company: Gedeon Richter
Product: Fylrevy
Active Ingredient: Estetrol/ Donesta
Disease: Hormone Replacement Therapy
Date: Jan 29, 2026
Shots:
- The CHMP has recommended 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); the decision will be anticipated 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, 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
Note:
- The following drugs have been approved; however, no PR was available:
- Inluriyo (Imlunestrant)
- GalenVita (Germanium (68ge) Chloride / Gallium (68ga) Chloride)
- Austedo (Deutetrabenazine)
- The following drugs have been granted CHMP; however, no PR was available:
- Supemtek (trivalent influenza vaccine)
- Kayshild (semaglutide)
- Ilumira (lutetium (177Lu) chloride)
Related Post: Insights+: EMA Marketing Authorization of New Drugs in December 2025
