Medical network May 4 - uniQure company's innovation gene therapy will soon come to an end, the therapy of bleak end is pegged to the super rare disease treatment in the field of other pharmaceutical companies offer lessons. 

 

After years of effort, in western countries, the first approved gene drug Glybera got a chance to business sales success, but now the uniQure announced plans to give up this super expensive treatments. UniQure said it would no longer request European authorities to extend the sales deadline for Glybera when sales authority expired in October. The gene therapy is priced at more than $1 million. 

 

nonmarket 

 

What's the problem? A lack of demand. Matthew Kapusta, chief executive of UniQure, said at a news conference that the use of the drug was extremely limited since it was approved in Europe in 2012. Even worse, UniQure expects demand for the drug to remain weak in the coming years. 

 

In addition, under the terms of the initial approval of Glybera, the company must pay some cash if it wants to delay sales. According to requirements, the biotech company to establish long-term care for the patient registration form, and complete a after-sales survey, set up the new risk management procedures, and to accept the annual review by the regulatory authorities. 

 

In addition, the company is required to maintain the pharmaceutical infrastructure and maintain communication with the regulators... It's all for an untouchable blockbuster! 

 

Last may, the MIT technology review magazine reported that Glybera for rare genetic disease, familial lipoprotein lipase deficiency, approved since 2012, only one patient received drug treatment. In order to get health insurance, the patient's doctor had to submit a thick sheet of text and call the CEO of a German insurance company. 

 

Glybera, in fact, are very effective in the treatment of: since the patient treatment, she has never been to a hospital, and in the use of Glybera, before her hospitalization dozens of times. 

 

Ultra rare disease drug pricing puzzle 

 

The city of Glybera also offers lessons for other pharmaceutical companies that develop treatments for super-rare diseases. Although the pharmaceutical companies have the ability to make those few select patients who are or have no choice but to benefit greatly, but the drug pricing must control within the scope of the can take on. 

 

Last year, GlaxoSmithKline (GlaxoSmithKline) gene therapy Strimvelis approved in Europe, for the treatment of severe combined immunodeficiency disease, this medicine is much cheaper than Glybera, priced at $665000, and GSK took out an invalid refund the security deposit. Super-expensive drugs are putting pressure on the medical budget, which is a potential way to pay for such drugs. 

 

Karp, starr said Glybera business after the uniQure company will transfer the energy to fight hemophilia B, the company plans to advance the project into the critical experimental stage. The company also plans to huntington's disease recruited subjects into further clinical proof of concept study, and bristol-myers Squibb (Bristol Myers Squibb) cooperation focus on cardiovascular disease. 

 

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If you had to pay for the most expensive prescription drug, it would cost you more than a ferrari 488 spider or a porsche 911 carrera each year. The most expensive drugs have two things in common: they are both used to treat rare and critically ill diseases, costing hundreds of thousands of dollars a year. 

 

NO. 1 Glybera 

 

Annual cost: $1 million 

 

Glybera (Alipogene tiparvovec) is a kind of gene therapy drug, can cure was diagnosed with a familial lipoprotein lipase deficiency (LPLD) in adult patients, this is a rare genetic disease, cause a lot of fat accumulation in the blood, can cause severe pancreatitis. Developed by Dutch drug company Uniqure, the drug costs up to $1 million a year. 

 

Glybera was approved by the European drug administration in 2012. Currently, americans can't get the drug directly, and the FDA requires additional clinical trials, and Uniqure decided not to carry out the trials further. 

 

NO. 2 Ravicti 

 

Annual cost: $794, 000 

 

Ravicti benzyl butyrate (glycerin) is a kind of treatment for congenital urea cycle disorder (UCD) medicines, it by eliminating ammonia, prevent the harmful accumulation of ammonia in the body and play a role. This accumulation of toxic substances can cause brain damage and death. The annual cost of each patient is nearly $794, 000, according to Horizon pharmaceuticals. 

 

In the United States, an estimated 2,000 people have urea cycle disorder. Ravicti can treat seven types of UCD, and whether Ravicti can safely and effectively treat n-acetylglutamate synthesis (NAGS) is a rare type of rare, inconclusive type of disease. 

 

NO. 3 Spinraza 

 

Annual cost: $750,000 

 

The spinal cord atrophy (SMA) drug, the drug Spinraza, is a new member of the most expensive prescription drug. It is an anti-oligonucleotide precursor compound that binds to the mutated RNA. 

 

The drug was originally developed by Ionis Pharmaceuticals, which was approved by the FDA on December 23, 2016. 

 

SMA is a rare, often fatal genetic disease that affects muscle strength and movement. Spinraza appropriate, is the first drug treatment costs $750000 in the first year, but the cost can drop to $375000 a year later, because the first year to more than the later years injection times. 

 

NO. 4 Lumizyme 

 

Annual cost: $626, 000 

 

Lumizyme (alglucosidase alfa, restructuring the Portuguese glycosidase alpha) is used to replace Pompey disease (Pompe diseases, Ⅱ glycogen storage disease of product) in patients with a loss or lack of enzyme in the body. Pompeii is a genetic disease caused by the accumulation of sugar in the body's cells called glycogen. The drug is listed by sanofi and costs more than $626, 000 a year. 

 

The incidence of Pompeii is estimated to be one in every 40,000 births, and the incidence of African americans is higher, about one in 14, 000. In 2006, sanofi's Myozyme was approved to treat Pompeii. However, when the company plans to expand the bioreactor of Myozyme, the FDA requires the approval process alone. That prompted Lumizyme's approval and listing in 2010. 

 

NO. 5 Carbaglu 

 

Annual cost: $585, 000 

 

Carglumic acid is used to treat patients with urea circulatory disorder (UCDs). Unlike Ravicti, however, Carbaglu specializes in the absence of n-acetyl-glutamate synthesis enzyme (NAGS). The drug's developer, Recordati, is pricing Carbaglu at no less than $585, 000 per patient per year. 

 

Carbaglu is the only drug approved by the FDA for the treatment of high amamemia, which is caused by a lack of patients in all age groups. There are seven types of UCD, although each type of patient is rare, but the absence of NAGS is the rarest. Because the diagnosis of NAGS is not enough, the actual number of people affected by UCD remains unknown. 

 

NO. 6 Actimmune 

 

Annual cost: $572,000 

 

Actimmune (Interferon gamma-1b, Interferon gamma-1b) can treat two rare genetic diseases: chronic granulomatosis (CGD) and severe malignant osteoporosis (SMO). Horizon pharmaceuticals is pricing Actimmune at $572, 000 per patient per year. 

 

The drug is an interferon gamma protein made by a biological method. The incidence rate of CGD in the United States is 1 per 200 000, while SMO has a rate of 1 person / 250 000. 

 

NO. 7 Soliris 

 

Annual cost: $543, 000 

 

Until recently, the world's most expensive drug, Eculizumab, was now in seventh place, with an annual cost of nearly $543, 000. 

 

Soliris treats two rare diseases: episodic night hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). The PNH destroys the patient's red blood cells and estimates that there are about 8, 000 to 10, 000 patients in North America and Europe. AHUS may lead to serious complications (including blood clot) affect the body's immune system, was diagnosed with the disease in patients with only 1500, but the world may be as many as 41000 patients with aHUS. 

 

conclusion 

 

The seven most expensive medicines are just the tip of the iceberg. According to 2015, more than 90 prescription drugs cost more than $100,000 a year. 

 

A rare disease is only for a limited number of patients, and it is right to share research and development and production and operating expenses. However, the inflated price of the rare disease has led to a lot of attention, and the United States has launched an investigation into the misuse of the rare drug act.