Dr. George D. Yancopoulos, the president and chief scientific officer at Regeneron, which, in partnership with Sanofi, makes the drug, said he expects the Food and Drug Administration to rule on dupilumab by March 29, 2017. The drug’s brand name will be Dupixent. The agency has given the drug breakthrough status, which provides expedited development and review of drugs for serious or life-threatening diseases.
Dr. Yancopoulos declined to speculate on dupilumab’s price, saying only that it will be “consistent with the value of the drug.” It is a biologic, the most expensive type of drug, and is injected every two weeks.
Atopic dermatitis experts said they have longed for a safe and highly effective treatment. In desperation, some prescribed other drugs off-label, like powerful immunosuppressants or high doses of steroids, which are far from ideal because even if they helped, their side effects can be severe — kidney failure with immunosuppressants, bone loss and even psychotic breaks with high-dose steroids.
Patients are miserable, Dr. Boguniewicz said. “Our patients and families haven’t slept through the night, not for days or weeks, but for months or years.”
Many doctors provide no treatments other than perhaps creams and ointments that do not stop the itching or soothe the red and weeping rash, said Dr. Jonathan I. Silverberg of Northwestern University’s Feinberg School of Medicine and a principal investigator in one of the studies.
Many sufferers can relate to the plight of the defense lawyer played by John Turturro in the HBO series “The Night Of.” He suffers from atopic dermatitis that started on his legs and his feet and later spread to his neck and head. Like so many patients, he tries treatment after treatment — bleach baths, covering the rash in Crisco and wrapping it with plastic wrap, steroids, Chinese medicine. He scratches it with chopsticks and disgusts people near him. But all to no avail.
Such experiences explain the excitement over the new drug, although researchers say they would like to see longer-term data.
“What we are seeing are some really impressive efficacy numbers,” Dr. Silverberg said. “But efficacy alone is not enough. It is the safety profile that is the real key. Everything we are seeing really looks great.”
Dr. Jon M. Hanifin, a professor of dermatology at Oregon Health and Science University and founder of the National Eczema Association, agreed. While not a principal investigator in the study, Dr. Hanifin did oversee the care of some patients enrolled in it.
“It’s wonderful,” he said. “We walk in the room and patients are smiling. These patients are the worst of the worst. Their life was destroyed.”
Dr. Yancopoulos was inspired in part to develop the drug because his father had severe atopic dermatitis, which he developed shortly after he got lung cancer at 70.
“More so than the cancer and the chemo, this rash and its horrible itch started dominating his life and ruining its quality,” Dr. Yancopoulos said. “Here’s a guy with Stage IIIB lung cancer — basically a death sentence — and he is more concerned and miserable about his skin and his itch.”
One participant in the trial, Lisa Tannebaum, a 53-year-old harpist in Stamford, Conn., was so thrilled that she wrote a letter to Regeneron suggesting they use her before and after photographs in advertisements. She developed a severe form of the disease 14 years ago and tried everything imaginable in conventional and alternative medicine without relief — specialized diets, immunosuppressive drugs, special clothing, bleach baths. She even had the gold fillings removed from her teeth on the theory that they may be causing an allergic response, but to no avail.
“It was like every day I had poison ivy and fire ants on myself,” she said. “You don’t sleep at all. You can’t go out, you have staph infections all the time,” because the skin’s protective barrier is broken by the rash. “I couldn’t drive my kids to school because the itching was so bad I couldn’t put my hands on the steering wheel.”
Now, she is performing again and will be playing her harp at Carnegie Hall on Oct. 30.
Herb Bull, 71, a retired Merck scientist in Westfield, N.J., had mild atopic dermatitis for years until three years ago, when it took a turn for the worse. The rash covered his entire body. Sleep was impossible, itching a constant torment. Even walking was difficult.
“He had weeping lesions all over his body,” said his doctor, Dr. Emma Guttman-Yassky, a principal investigator in the trial and professor of dermatology and immunology at Mount Sinai School of Medicine.
“I thought I might as well give up and die,” Mr. Bull said.
It took months for the drug to work, he said, but when it did, the change was miraculous. His rash and the itching went away.
The new drug, Mr. Bull said, “saved my life.”