In the United States, the FDA has authorized a drug capable of slowing the evolution of diabetes 1 in both adults and children. All the details about the therapy, who produces it and how much it will cost
The US Food and Drug Administration (Fda) has approved Tzield (teplizumab-mzwv), the first drug, co-produced by Provention Bio and Sanofi, capable of slowing the onset of stage 3 type 1 diabetes in adults and in children at risk.
Diabetes 1 is an autoimmune form in which the immune system damages the portion of the pancreas responsible for producing insulin.
WHO WILL RECEIVE IT
The FDA has authorized the use of this new therapy for ages 8 and older for patients with stage 2 type 1 diabetes to slow the onset of stage 3 diabetes.
The green light for Tzield "adds an important new treatment option for some at-risk patients," said John Sharretts, director of the Division of Diabetes, Lipid Disorders, and Obesity at the FDA's Center for Drug Evaluation and Research. "The drug's potential to delay the clinical diagnosis of type 1 diabetes may offer patients months or years without the burden of disease."
In clinical trials,Tzield delayed progression by just over two years, but the benefits lasted much longer in some of the study participants.
The safety and efficacy of Tzield were evaluated in a randomized, double-blind study of 76 patients with stage 2 type 1 diabetes.
During the trial, some patients received the drug and others a placebo once a day by intravenous infusion for 14 days.
Follow-up found that 45% of 44 patients who received Tzield developed stage 3 type 1 diabetes later than 72% of 32 patients who received a placebo.
The median time from randomization to diagnosis was 50 months for patients who received Tzield and 25 months for those who received a placebo. This represents a statistically significant delay in the development of stage 3 type 1 diabetes.
HOW DOES IT WORK
Tzield, the FDA note reads, binds to certain cells of the immune system and delays the progression to stage 3 type 1 diabetes. It is able to deactivate immune cells that attack insulin-producing cells, while increasing the percentage of cells that help moderate the immune response.
The drug, as in the trial, is administered by intravenous infusion once a day for 14 consecutive days.
WHO PRODUCES IT
HOW MUCH WILL IT COST
According to CNN , which reported on a talk between the manufacturer and investors, the production cost of a full course of treatment will be around $194,000, but this is not expected to be the price consumers will pay.
TYPE 1 DIABETES
Type 1 diabetes, explains the FDA, is a disease that occurs when the immune system attacks and destroys cells that produce insulin. People with this disease have high glucose levels that require insulin injections (or the use of an insulin pump) to survive, and they need to check their blood sugar levels regularly throughout the day.
Although type 1 diabetes can appear at any age, it is usually diagnosed in children and young adults. A person is more at risk if they have a parent or brother or sister who have it, but most patients typically have no family members with type 1 diabetes.
In 2019, approximately 1.9 million people had type 1 diabetes in the United States, according to the American Diabetes Association , including 244,000 children and adolescents.
WHY IT IS IMPORTANT TO SLOW DOWN THE DISEASE
“The onset of stage 3 type 1 diabetes is a life-changing moment: once insulin-producing cells are no longer able to maintain normal glycemic control, this irreversible condition can lead the patient to have to , in just one year, 1,460 finger pricks to control blood glucose levels, approximately 1,100 insulin injections and experience an average of 127 hypoglycemia episodes,” explained Eleanor Ramos, chief medical officer of Provention Bio.
This is a machine translation from Italian language of a post published on Start Magazine at the URL https://www.startmag.it/sanita/la-fda-ha-approvato-un-farmaco-che-ritarda-evoluzione-del-diabete-1/ on Thu, 24 Nov 2022 06:47:59 +0000.