Glybera - A medicine for the rich only

Glybera gene therapy helps treat rare diseases and has yielded very positive results. However, it is not widely applied because the cost is too expensive, up to 1 million USD (about 22 billion VND) for the whole treatment process.

The drug we are talking about is Alipogene tiparvovec , better known as Glybera - that is praised by the West as the " first human gene therapy " that can modify your DNA. the patient is directed against a particular disease.

In 2012, the European Commission (EC) approved the sale of Glybera gene therapy in the European and North American markets to treat Lipoprotein Lipaza disorder - a rare form of human disease. It is estimated that this treatment method costs about 1 million USD for one treatment.

Last fall, when Dr. Elisabeth Steinhagen-Thiessen wanted to use Glybera to treat her patients, things didn't go smoothly. Elisabeth said she had to prepare a thick file like a dissertation to submit to the German Administration and then she had to personally call the German Health Insurance Fund (Deutsche Angestellten-Krankenkasse - DAK ) to request support of $ 1 million in treatment costs.

This drug is also called Alipogene tiparvovec.

Earlier, in September last year, Elisabeth injected 40 doses of Glybera into the muscles of a 43-year-old woman suffering from an extremely rare disease, a genetic disorder caused by a lack of Lipoprotein Lipase (Lipoprotein Lipaza Deficiency - enzyme). LPLD). Without this enzyme, the patient's body will not be able to break down fat from digested food, resulting in fat particles called chylonmicrons accumulating in the blood. Symptoms of this disease include: abdominal pain, nausea, loss of appetite . And this miserable woman has been hospitalized up to 40 times! In this case, Glybera plays a role in preventing the metabolism of fat molecules fixed into the blood sugar. Dr. Elisabeth said that the treatment was successful beyond imagination. Currently, this woman did not have to return to the hospital but lived a normal life after receiving Glybera therapy.

In 2013, UniQure - a successful research firm Glybera gave up its plan to apply for a license in the US but moved to the European market to sign a cooperation agreement with Chiesi Farmaceutici for exclusive commercialization of Glybera. UniQure CEO said: " I think we have learned a lot of things, know what we should do and should not do. However, the company is still suffering a lot of losses." Currently, UniQure is focusing on developing other gee therapies, including the gene for treating haemophilia.

In fact, Glybera treatments are thought to be failures of medicine and are a loss-making product for UniQure, although this is an effective treatment for rare cases. A single Glybera dose can change the genetic direction inside human cells for many years, even for a lifetime and help save lives, but the problem is not merely therapeutic efficacy. but also in the ability to respond to the patient's payment. The price of 1 million USD is too high and only suitable for groups of patients in developed countries (wealthy group), but for poor countries this is really a problem.

Is there any alternative?

In addition to Glybera, there is at least one more gene therapy called Imlygic approved in China that allows the treatment of cancer by directly injecting it (a form of genetic modification of herpes virus) into block tissue. u aim to destroy cancer cells from within. According to the study, about 16% of patients who received this injection had a reduced tumor. Late last year, Imlygic was approved by the US to treat skin cancer. Gene therapy is still promising to treat reverse genetics and treatments will soon be commercialized.

In April, the European government gave the green light for the implementation of gene therapy called Strimvelis, which helps treat severe immunocompromised deficiency eggs, sold by Glaxo SmithKline.

A single Glybera dose can change the genetic direction inside human cells for many years.

And by 2017, Spark Therapeutics - a Philadelphia company may be able to win US approval of new gene treatments that help blind people see sunlight.

What is special is that these diseases are very rare. Glaxo estimates that only 14 cases of severe combined immunodeficiency were detected in Europe.

Because these are rare diseases that require expensive technology treatment, they push the prices of these drugs to unprecedented highs. Analysts said that, if treated according to the method of Spark Therapeutics, patients will have to pay up to $ 500,000 (about 11 billion) for each eye. Currently, 670 people are being treated for this gene, of which 68 are in the final stage (stage 3) and the urgent need to be clarified is how much it will be paid. Come on.

The CEO at Glaxo said that the company will not price its drugs up to $ 1 million like Glybera."We are trying to create a balance between nurturing creativity and creating value for the health care system," he said. He also added: "I know there are many people interested in the first gene therapy in Europe (Glybera) that sells for about $ 1 million and I can be certain that Strimvelis will be low cost. significantly more ".

Clearly, the Glybera approved event used in 2012 is an extremely important milestone, suggesting that gene therapy - once thought to be a potentially dangerous and risky cure, is now safe. Full and ready for commercialization. Since then, many new pharmaceutical companies have been established and received public attention. Investment bank Piper Jaffray said that 2015 was the most important year of gene therapy when about $ 2 billion was used to subsidize 10 gene therapy companies in the United States.