EU regulators back a drug that delays type 1 diabetes and a first gene therapy for WAS
The CHMP's November meeting produced ten positive opinions. Two of them — teplizumab and a Telethon-sponsored gene therapy — address conditions the EU has had no authorised treatment for.
| Group | Value (%) |
|---|---|
| Teplizumab | 45 |
| Placebo | 72 |
The European Medicines Agency's Committee for Medicinal Products for Human Use recommended ten new medicines for approval at its 10–13 November meeting, publishing the outcome on 14 November [s1]. It issued no negative opinions on new medicines, recommended extensions of indication for four already authorised products — Koselugo, Minjuvi, Veyvondi and Xerava — and saw three initial applications withdrawn [s1].
A CHMP positive opinion is a scientific recommendation, not an authorisation. The European Commission issues the binding decision afterwards, and pricing and reimbursement are then settled member state by member state [s2].
Two of the ten opinions are first-of-kind.
Delaying, not treating, type 1 diabetes
The committee adopted a positive opinion for Teizeild (teplizumab), from Sanofi Winthrop Industrie, to delay the onset of stage 3 type 1 diabetes in adults and in children from eight years of age who have stage 2 disease [s1].
The framing matters. Type 1 diabetes progresses through three stages, and symptoms — and the need for daily insulin — normally appear at stage 3 [s2]. Teplizumab is an antibody that slows the pace at which the immune system destroys pancreatic beta cells, and it is given as an intravenous infusion once daily for 14 consecutive days [s2]. It does not cure anything. An estimated 2.2 million people in the EU live with type 1 diabetes, and there are currently no authorised treatments to delay or cure it [s2].
The recommendation rests on a randomised, double-blind, placebo-controlled trial in 76 patients with stage 2 type 1 diabetes [s2]. Median time to developing stage 3 disease was 50 months in patients given teplizumab and 25 months in patients given placebo [s2]. Over a median follow-up of 51 months, 20 of 44 teplizumab-treated patients (45%) developed type 1 diabetes, compared with 23 of 32 placebo patients (72%) [s2]. Results from several other studies show teplizumab significantly preserved pancreatic beta-cell function against placebo [s2].
Seventy-six patients is a small trial, and the primary result is a delay measured in months rather than a change in eventual outcome — most treated patients in the trial still progressed. The most common side effects were low levels of various white blood cells (lymphocytes, leucocytes and neutrophils) and rash; the most frequent serious adverse reaction, reported in 2% of patients, was cytokine release syndrome, which causes fever, vomiting, shortness of breath, headache and low blood pressure [s2]. EMA notes that the product information and risk management plan include risk mitigation measures [s2].
Teizeild was supported through EMA's PRIME scheme, having been granted eligibility on 17 October 2019 [s2].
A gene therapy from a non-profit developer
The committee also adopted a positive opinion for Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, sponsored by Fondazione Telethon Ets and carrying orphan designation [s1].
Wiskott-Aldrich syndrome is a rare inherited disease, seen almost exclusively in males, caused by abnormalities in the gene producing the WAS protein [s3]. Without functional WAS protein, blood and immune cells do not develop normally: patients bruise and bleed easily because they have too few normal platelets, get frequent infections that can progress to sepsis, and carry a higher risk of some cancers including lymphoma [s3]. Patients with a compatible donor are treated by haematopoietic stem cell transplantation; for most patients without one, there has been an unmet need [s3]. The recommended indication is for people aged six months and older with a WAS gene mutation for whom transplantation is appropriate but no suitable donor is available [s3].
The therapy collects the patient's own CD34+ stem cells, genetically modifies them ex vivo with a lentiviral vector encoding the human WAS protein, and returns them by a single intravenous infusion after a conditioning regimen [s1][s3].
The evidence base is 27 patients: a single-arm main study in ten children aged one to nine, supported by another clinical trial and an expanded access programme covering 17 patients aged one to 35 [s3]. The annualised rate of severe infections fell from 2.0 events in the 12 months before treatment to 0.15 events at one to two years afterwards and 0.12 events at two to three years [s3]. The annualised rate of moderate and severe bleeding episodes fell from 2.0 events to 0.16 events at two to three years [s3]. The most common side effects were attributed to the procedures and medicines needed to deliver the therapy — the conditioning regimen, pre-treatment, and administration site problems such as infusion device infections and catheter site bleeding [s3].
This is a single-arm, before-and-after comparison in a very small cohort, which is standard for gene therapies in ultra-rare disease and remains a weaker design than a controlled trial. EMA's Committee for Advanced Therapies judged the benefits to outweigh the risks in the defined population, and the CHMP agreed [s3]. Waskyra was developed with support from an EMA pilot offering enhanced regulatory help to academic and non-profit developers of advanced therapy medicinal products [s3].
The rest of the meeting
The other eight opinions covered Dawnzera (donidalorsen), an orphan medicine for routine prevention of recurrent hereditary angioedema attacks in people aged 12 and over; GalenVita, a radionuclide generator for PET imaging; Inluriyo (imlunestrant) for ESR1-mutated advanced or metastatic breast cancer; VacPertagen, an acellular pertussis vaccine; a hybrid enzalutamide for prostate cancer; biosimilar insulin glargine and denosumab; and generic teduglutide for short bowel syndrome [s1].
Three applications were withdrawn: an insulin aspart injection, Nurzigma (pridopidine) for Huntington's disease, and Ohtuvayre (ensifentrine) for COPD maintenance treatment [s1]. On re-examination, the committee confirmed its earlier recommendation not to treat levacetylleucine (Aqneursa) as a new active substance, and the holder of Rezurock (belumosudil) has requested re-examination of the negative opinion adopted at the October meeting [s1].
Running totals for 2025 stand at 97 positive opinions on new medicines, six negative opinions, 77 positive opinions on extensions of indication, and 21 withdrawn applications [s1].
Sources
- [s1] Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 10-13 November 2025, European Medicines Agency, 14 November 2025. https://www.ema.europa.eu/en/news/meeting-highlights-committee-medicinal-products-human-use-chmp-10-13-november-2025
- [s2] First-in-class treatment to delay onset of type 1 diabetes, European Medicines Agency, 14 November 2025. https://www.ema.europa.eu/en/news/first-class-treatment-delay-onset-type-1-diabetes
- [s3] First gene therapy to treat rare disease Wiskott-Aldrich syndrome, European Medicines Agency, 14 November 2025. https://www.ema.europa.eu/en/news/first-gene-therapy-treat-rare-disease-wiskott-aldrich-syndrome
Sources
- Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 10-13 November 2025 — European Medicines Agency , November 14, 2025
- First-in-class treatment to delay onset of type 1 diabetes — European Medicines Agency , November 14, 2025
- First gene therapy to treat rare disease Wiskott-Aldrich syndrome — European Medicines Agency , November 14, 2025
More on
A funding change moved 98 NZ children off their insulin pumps. No control arm
PHARMAC's October 2024 decision left one brand's sensors unfunded, forcing a device switch. A single-centre audit of 79 children found HbA1c fell slightly — and cannot say the switch caused it.
EU regulators back a bladder cancer gene therapy and two lung cancer drugs
The European Medicines Agency's March meeting produced five positive opinions, three of them for orphan-designated cancer and rare-disease treatments already available in some other markets.
Norway vaccinated a generation against rotavirus. Type 1 diabetes did not fall.
A 740,744-child natural experiment tested a decade-old hypothesis that rotavirus vaccine might prevent type 1 diabetes. It found no protective effect — and no evidence of harm either.
A glucose-monitor study reached for a hard outcome — death — and found a modest signal
Emulating a trial inside Veterans Health Administration records, researchers estimated adjusted mortality risk ratios of 0.90 to 0.84 for starting continuous glucose monitoring in older-onset type 1 diabetes.