F.D.A. Approves drug that can delay type 1 diabetes

The therapy will cost nearly $200,000. But it's the first treatment in 100 years to alter the course of the disease, which typically begins in adolescence.

The Food and Drug Administration has approved the first treatment that can delay - perhaps for years - the onset of type 1 diabetes, a disease that often appears in adolescents.

The new drug, teplizumab, is manufactured by Provention Bio, which will partner with Sanofi to market the drug in the United States under the brand name Tzield. In an investor call on Friday, Provention said the drug will cost $13,850 per vial or $193,900 for the 14-day course. The company said teplizumab should be available by the end of the year.

The drug, which the F.D.A. approved on Thursday, does not cure or prevent type 1 diabetes. Instead, it delays its onset for an average of two years and, for some lucky patients, much longer - the longest to date is 11 years, said Dr. Kevan Herold of Yale, principal investigator in the drug's trials.

The only other treatment for the disease, insulin, was discovered a 100 years and does not affect disease progression. It simply replaces what is missing.

Teplizumab will be used to treat patients at high risk of type 1 diabetes who have antibodies that indicate an immune attack on their pancreas and whose blood sugar tolerance is not normal. Treatment involves a 14-day infusion of the drug, a monoclonal antibody that blocks T cells from attacking insulin-producing cells in the pancreas.

" Talk to anyone with type 1 diabetes and every day that you don't have to test your blood sugar four times a day and inject yourself with insulin is a glorious day,” said Dr. Mark S. Anderson , director of the Diabetes Center at the University of California, San Francisco, and investigator for the pivotal clinical trial that led to the treatment's approval. Dr. Anderson has served as a paid consultant for Provention in the past.

Dr. John Buse, a diabetes expert at the University of North Carolina who was not involved in the study, called the approval "really exciting. and said she would "turn the type 1 diabetes world upside down".

"There is t Always had an idea that screening would be a good idea," he said. But medical experts "have never really promoted it for detecting type 1 diabetes."

It won't be easy — no screening test will 'is. But in this case, very few people screened will have this rare but serious disease, which affects only four out of 1,000 people in the general population, or 1.4 million Americans.

Type 1 Diabetes usually appears in adolescence when patients are suddenly tired all the time, urinate frequently, drink large amounts of water and lose weight.

With a diagnosis of diabetes, their lives are completely changed. They must measure their blood sugar and take insulin for the rest of their lives. Each time they eat a meal, they have to calculate how much insulin they need. If they take too much, they may pass out or have a seizure or even end up in intensive care.

They also face the specter of complications - an illness eye to blindness, kidney failure, heart disease and stroke. Without good blood sugar control, complications can occur as early as five years after diagnosis, Dr. Anderson said.

The new treatment, Dr. Anderson said, "opens the door," in the same way that the first cancer immunotherapy was a breakthrough into a new era of treatment about a decade ago. He expects that as diabetes immunotherapy improves, the disease can be stopped before it can set in.

The new drug is not a cure for the much older type of diabetes common, Type 2, in which the pancreas makes insulin but the body's cells do not respond to it.

The history of the new treatment dates back to the years 1988...