Monday December 9, 2019

Most Expensive Medicine Treats Rare Inherited Disease for $2M

The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called spinal muscular atrophy

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expensive medicine
FILE - The logo of Swiss pharmaceutical company Novartis. VOA

U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby’s muscle control and kills nearly all of those with the most common type of the disease within a couple of years.

The treatment is priced at $2.125 million. Out-of-pocket costs for patients will vary based on insurance coverage. The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called spinal muscular atrophy. The treatment targets a defective gene that weakens a child’s muscles so dramatically that they become unable to move, and eventually unable to swallow or breathe. It strikes about 400 babies born in the U.S. each year.

The Food and Drug Administration on Friday approved the treatment, called Zolgensma, for all children under age 2 who are confirmed by a genetic test to have any of the three types of the disease. The therapy is a one-time infusion that takes about an hour.

Novartis said it will let insurers make payments over five years, at $425,000 per year, and will give partial rebates if the treatment doesn’t work.

The one other medicine for the disease approved in the U.S. is a drug called Spinraza. Instead of a one-time treatment, it must be given every four months. Biogen, Spinraza’s maker, charges a list price of $750,000 for the first year and then $350,000 per year after that.

expensive medicine
FILE – This Oct. 14, 2015, photo shows the Food and Drug Administration campus in Silver Spring, Md. VOA

‘Dramatically transforms’ lives

The independent nonprofit group Institute for Clinical and Economic Review, which rates the value of expensive new medicines, calculated that the price of the new gene therapy is justifiable at a cost of $1.2 million to $2.1 million because it “dramatically transforms the lives of families affected by this devastating disease.”

ICER’s president, Dr. Steven D. Pearson, called the treatment’s price “a positive outcome for patients and the entire health system.” The defective gene that causes spinal muscular atrophy prevents the body from making enough of a protein that allows nerves that control movement to work normally. The nerves die off without the protein.

In the most common type, which is also the most severe, at least 90% of patients die by age 2, and any still alive need a ventilator to breathe. Children with less-severe types become disabled more slowly and can live for up to a couple decades.

Zolgensma works by supplying a healthy copy of the faulty gene, which allows nerve cells to then start producing the needed protein. That halts deterioration of the nerve cells and allows the baby to develop more normally.

In patient testing, babies with the most severe form of the disease who got Zolgensma within 6 months of birth had limited muscle problems. Those who got the treatment earliest did best. Babies given Zolgensma after six months stopped losing muscle control, but the medicine can’t reverse damage already done.

expensive medicine
The medicine, sold by the Swiss drugmaker Novartis, is a gene therapy that treats an inherited condition called spinal muscular atrophy. Pixabay

Success story

Evelyn Villarreal was one of the first children treated, at eight weeks. Her family, from Centreville, Va., had lost their first child to spinal muscular atrophy at 15 months. Two years later when Evelyn was born a test showed she also had the disease, so the family enrolled her in the gene therapy study at Nationwide Children’s Hospital in Columbus, Ohio.

Evelyn is now 4½ years old and showing no muscle problems other than minor trouble standing up, said her mother, Elena Villarreal. She has been feeding herself for a long time, she draws and speaks well, and will be starting kindergarten in the fall. “She’s very active and goes to the playground a lot,” said Elena Villarreal. “She’s walking and even jumping.”

ALSO READ: WHO Chief Emphasizes the Importance of Universal Health Coverage

It is too early to know how long the benefit of the treatment lasts, but doctors’ hopes are rising that they could last a lifetime, according to Dr. Jerry Mendell, a neurologist at Nationwide Children’s. Mendell led one of the early patient studies and is Evelyn’s doctor. “It’s beginning to look that way,” he said, because a few children treated who are now 4 or 5 still have no symptoms.

Early diagnosis is crucial, so Novartis has been working with states to get genetic testing for newborns required at birth. It expects most states will have that requirement by next year. The FDA said side effects included vomiting and potential liver damage, so patients must be monitored for the first few months after treatment. (VOA)

Next Story

Resistance to Antibiotics Rise Among Indian Patients in Pace

There is increase in resistance to antibiotics including clarithromycin which is undoubtedly a worrisome situation in the country

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Antibiotics
In India, a huge population prefers to consume over-the-counter (OTC) drugs without even consulting a doctor. In the long run, this may make them resistant to most of the Antibiotics including clarithromycin. Pixabay

Resistance to commonly-used Antibiotics like clarithromycin is rising among Indian patients and that too at quite a fast pace, health experts have warned.

Clarithromycin is used to treat a wide variety of bacterial infections. This medication can also be used in combination with anti-ulcer medications to treat certain types of stomach ulcers.

According to the World Health Organization (WHO), antibiotic resistance is one of the biggest threats to global health, food security and development today.

Sunil Sofat, Additional Director, Department of Interventional Cardiology (Adult) at Jaypee Hospital in Noida, said that every antibiotic medicine has its own mechanism to treat diseases.

“Yes, this is true that the resistance to clarithromycin is rising among the Indian patients and that too at quite a fast pace. There are multiple factors for the same but one of the major reasons behind it is self-medication,” Sofat told IANS.

“In India, a huge population prefers to consume over-the-counter (OTC) drugs without even consulting a doctor. In the long run, this may make them resistant to most of the antibiotics including clarithromycin,” Sofat added.

Antibiotics
Resistance to commonly-used Antibiotics like clarithromycin is rising among Indian patients and that too at quite a fast pace, health experts have warned. Pixabay

In a recent study presented at United European Gastroenterology (UEG) Week Barcelona 2019, researchers have found that resistance to clarithromycin, one of the most established antimicrobials used to eradicate Helicobacter pylori (H. pylori), had increased from 9.9 per cent in 1998 to 21.6 per cent last year, with increases in resistance also seen for levofloxacin and metronidazole.

The study, which analysed 1,232 patients from 18 countries across Europe, investigated resistance to antibiotics regularly taken for Helicobacter pylori infection, a harmful bacterium associated with gastric ulcers, lymphoma and gastric cancer.

According to Gaurav Jain, Senior Consultant, Internal Medicine at Dharamshila Narayana Superspeciality Hospital, New Delhi, antibiotic resistance is a major concern.

“In India the consumption of antibiotics without consulting a qualified physician is quite common which is leading to its resistance,” Jain told IANS.

“There is increase in resistance to antibiotics including clarithromycin which is undoubtedly a worrisome situation in the country,” Jain said.

However, Deepak Verma of Internal Medicine at Columbia Asia Hospital, Ghaziabad said: “Most of the cases that we see in India are connected to gram-negative bacteria such as e.coli that causes urinary tract infection (UTI).”

He added that the main causes for antibiotic resistance in India are its rampant misuse where people indulge in self-doctoring as well as taking medicines prescribed by unregistered medical practitioners, including quacks who suggest antibiotics quite indiscriminately.

Antibiotics
According to the World Health Organization (WHO), resistance to Antibiotics is one of the biggest threats to global health, food security and development today. Wikimedia Commons

“They primarily use antibiotics symptomatically which is not a correct method for all ailments — without blood and urine culture. Antibiotics can force the pathogen to develop resistance,” Verma explained.

ALSO READ: Researchers Develop a New Vaccine to Stop Bovine TB

“Since the clinical culture in India is different from that of the western countries, the lack of awareness of the right process to prescribe antibiotics increases the chances of people using antibiotics without questioning,” he stressed. (IANS)