Sanofi's type 1 diabetes drug poised for EU rollout

On World Diabetes Day, Sanofi is celebrating an approval recommendation in the EU for Teizeild, a first-in-class drug designed to delay the progression of type 1 diabetes (T1D).

The EMA's human medicines committee, the CHMP, has recommended anti-CD3 antibody Teizeild (teplizumab) for use in patients aged eight and over with stage 2 T1D in order to delay the progression to stage 3.

Stage 1 T1D is when the immune system starts attacking insulin-producing cells in the body, while stage 2 marks the point at which blood glucose levels start to become abnormal, but patients still have no symptoms. In stage 3, clinical symptoms like excessive thirst, frequent urination, weight loss, and fatigue start to occur.

Sanofi acquired Teizeild when it bought Provention Bio for $2.9 billion in 2023, a few months after the drug was approved by the FDA under the Tzield brand name. Sales of the drug in the US have been slow to take up, but more than doubled to €54 million last year, adding another €47 million – up by a third – in the first nine months of this year.

It has also recently been approved in other markets, including China and the UK, and analysts have suggested that, in time, it could become a $1 billion-a-year brand.

The CHMP's positive opinion means that EU approval should be forthcoming within the next few weeks, and if that does occur, Teizeild will be the bloc's first disease-modifying therapy for T1D. An estimated 2.2 million people in the EU live with this type of diabetes.

Teplizumab delays disease progression by reducing the pace of the patient's self-destruction of pancreatic beta cells. It is administered by intravenous infusion once daily for 14 consecutive days.

In the TN-10 study that supported the recommendation, the antibody delayed the onset of stage 3 T1D by a median of approximately two years compared to placebo, and the proportion of patients who remained in stage 2 T1D was twice as high with Teizeild at 57% versus 28% of the control group.

Wiskott-Aldrich syndrome gene therapy

Meanwhile, in another EU first, the CHMP also backed approval of Fondazione Telethon ETS' Waskyra (etuvetidigene autotemcel), which could become the first genetic therapy for patients with Wiskott-Aldrich syndrome (WAS), a rare, inherited disease that causes bleeding and immune suppression. It mainly affects boys and has an estimated incidence of 1 in 250,000 male births.

Waskyra is a one-shot therapy made from stem cells collected from patients' blood and modified to create functional copies of the WAS gene, which is mutated in the disorder. It is also under regulatory review in the US.

Other new therapeutics recommended at the CHMP's November meeting included Otsuka/Ionis' plasma prekallikrein (PPK) inhibitor Dawnzera (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema, and Eli Lilly's oral selective oestrogen receptor degrader (SERD) Inluriyo (imlunestrant) for locally advanced or metastatic breast cancer with a specific mutation in the gene ESR1.

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