Friday June 21, 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

Ayurveda Brings To You The Science of Natural Cure

The other regimens to be followed will add to its benefit when consulted by a doctor as the series of pros-cons checklist is assessed with your constitution and the daily habits.

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An Ayurvedic practitioner applying oils using head massage.. Hindu Council

BY: Naveen Shukla, Dr Vishal Sharma, Dr Nikhila Venugopal. Nature Care Ayurveda

Ayurveda, the offspring of Atharvaveda and appreciated as the fifth Veda is the holistic system of medicine to promote health and to prevent diseases. This wonderful science has put up the Indian culture into great heights and is still a recollection of the cultural heritage. The powerful logic and philosophy which provides a link between the living and non living matters of the universe, is still appreciable in the current era .This Indian system of medicine has laid principles and methods of treatment for the chronic illnesses where there is no definite curative treatment and symptomatic relief is the only existing treatment solution.

The beauty of Ayurveda lies in the factor that when a patient comes to an Ayurvedic doctor the root cause or the Nidhana is being focused leading to the pathology and pathogenesis. This systematic modality of how beautiful a disease is being portrayed and it’s treatment accordingly is the greatest achievement in curing the chronic illness.
Ayurveda gives importance and corrects the improper food habits, suppression of natural urges, irregular daily regimen, seasonal regimen; psychological factors like fear stress anxiety which is a striking factor in a disease manifestation.

Being a whole body and mind oriented science, Ayurveda focuses on almost all of the bodily functions associated with heat, and its imbalances, nervous imbalance, functions of digestive tract and also controls structure, metabolism, and fluid balance of the body.
Ayurveda is gaining its importance and value because of its therapeutic value too. In the olden days, man lived very close to nature and cured himself by the materials from the nature which were considered to be the potent herbs which can do wonders as it possess infinite potencies.

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The commonly used spices in most kitchen like cumin seeds, fennel seeds, cinnamon, nutmeg, cloves, cardamom etc are known to pacify the overpowered Vata. Pixabay

The total balance of the environment, mind, body and spirit promotes Ayurveda achieve its goal towards a complete human wellness. As explained, the season and the regimens followed play an indispensable role in relation to the human body as Ayurveda considers that each season has its own value due to the dominance and aggravation of the specific dosha.

This autumn also provides some recommendation commencing from choosing a particular food and daily activity to balance the bodily transition and seasonal change. Being governed by pitta and vata, steps must be taken to keep them in balance. Foods that promote warmth and increases the moisture content of the body is highly appreciated.

Ayurveda herbs
Ayurveda herbs – Thomé Salix alba clean
Hindu Council

The commonly used spices in most kitchen like cumin seeds, fennel seeds, cinnamon, nutmeg, cloves, cardamom etc are known to pacify the overpowered Vata. Health is at its best during the Autumn, if oily, raw and junk foods are avoided as it hampers the digestive tract creating to many illness. Warm fluid does provide a soothing reaction to the sluggish digestive tract.

Also Read: Hindu Icons Which Have Spiritual Significance

The other regimens to be followed will add to its benefit when consulted by a doctor as the series of pros-cons checklist is assessed with your constitution and the daily habits. Thus the autumn gifts you an individualized healthy package of the right diet and lifestyle.

Roots of Ayurveda are so deep that its ultimate aim is a healthy body, mind and longevity. Efforts have been constantly put to elevate and preserve the great science of life- Ayurveda. We can proudly say Ayurveda as our heritage and pride which will keep flowing on to generations.with this sole aim of promoting a good health. (Originally Published: Hindu Council of Australia)