Heavy! The world's first new drug to delay type 1 diabetes was approved by the FDA

Today, the U.
S.
Food and Drug Administration (FDA) announced that it has approved Teplizumab, a drug developed by Provention Bio Teplizumab-MZWV injection is used to delay the onset of
stage 1 diabetes in adults currently in stage 3 diabetes and in stage 2 diabetes patients aged 8 years and older.

John, director of the Division of Diabetes, Lipid Disorders and Obesity at the FDA's Center for Drug Evaluation and Research Dr.
Sharretts said: "Today's approval of a homogeneous therapy adds an important new treatment option
for certain high-risk patients.
The drug has the potential to delay the clinical diagnosis of type 1 diabetes and allow patients to be free of the burden
of the disease for months to years.
”

Type 1 diabetes is an autoimmune disease in which the immune system attacks and destroys insulin-producing cells, resulting in insulin deficiency and impaired
blood sugar regulation.
Because blood sugar levels are elevated in people with type 1 diabetes, insulin injections (or wearing an insulin pump) are needed to survive, and blood sugar levels
must be checked regularly daily.
Although type 1 diabetes can develop at any age, it is usually higher in children and young adults, and a person's risk of developing type 1 diabetes is higher if they have a parent, brother or sister, although most people with type 1 diabetes do not have a family history
.

Teplizumab is a monoclonal antibody that targets CD3 antigen on the surface of T cells, and by binding to CD3 on the surface of effector T cells, it inhibits the attack of T cells on islet β cells, thereby protecting islet β cells from destruction and delaying the progression of
the disease.
In addition, Teplizumab inactivates immune cells that attack islet β cells while increasing the proportion
of cells that regulate immune responses.
Teplizumab is given intravenously once a day for 30 minutes for 14 days
.

The approval is based on a randomized, double-blind, event-driven, placebo-controlled trial of 76 patients with stage 2 diabetes who evaluated the safety and efficacy
of Teplizumab.
In the trial, patients were randomized to receive either Teplizumab or placebo with a once-daily intravenous infusion for 14 days
.
The primary measure of efficacy was the time from
randomization to diagnosis of stage 3 type 1 diabetes.

The trial results showed that during a median follow-up of 51 months, 45 percent of the 44 patients treated with Teplizumab were later diagnosed with stage 3 type 1 diabetes, compared with 72 percent of the 32 patients treated with placebo who were diagnosed with stage 3 type 1 diabetes
.
The intermediate time from randomization to diagnosis of stage 3 type 1 diabetes was 50 months in the Teplizumab group and 25 months
in the placebo group.
This suggests a statistically significant delay
in delaying the progression of Teplizumab to stage 3 type 1 diabetes.

In addition, Teplizumab treatment reversed the decline in C-peptide levels, suggesting that it improved the function of pancreatic islet β cells, including the ability to
secrete insulin.
In terms of safety, the most common side effects of Teplizumab include decreased levels of certain white blood cells, rash, and headache, which are safe and well tolerated
.

Teplizumab's road to development and go-to-market has been a tumultuous one
.
On the road to research and development, from the initial development by MacroGenics, to the abandonment of Eli Lilly after the introduction of $41 million, to Provention Bio introduced, Teplizumab finally made significant progress in 2019 and was granted breakthrough therapy designation
by the FDA.
In terms of marketing, Teplizumab submitted for marketing authorization on a rolling basis in 2020 and obtained priority review qualification in 2021, but was rejected by the FDA due to pharmacokinetic comparability issues; In early 2022, Provention Bio submitted the application again, and six months later, the FDA's review of the drug was postponed for three months
due to the review of major amendments.
Today, Teplizumab has finally been approved for listing
.

Although the road to R&D and market launch is long, it is worth waiting to bring better results to patients
.

Source: FDA official website

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