FInally Gene Therapy Drug is for sale

The western world’s first gene therapy drug is set to go on sale in Germany, with a price tag that could amount to an £870,000 cost to treat a single patient.

Glybera, a treatment for the rare genetic condition lipoprotein lipase deficiency (LPLD), which clogs the blood with fat, has been developed by Dutch biotech firm UniQure and Italian marketing marketing partner Chiesi. It is undergoing an assessment of benefits by Germany’s federal joint committee, which will report by April 2015.

But the company is seeking a retail price of €53,000 (£42,000) per phial, which equates to €1.1m (£870,000) for a course of treatment for a typical LPLD patient. This price will be subject to a discount under Germany’s drug pricing system.

A Chiesi spokeswoman confirmed the launch price and added that a final figure would be set after the German authorities gave their verdict and negotiations are held with health insurance funds. “First commercial treatments are expected in the first half of 2015,” she said.

UniQure, which will get a net royalty of between 23% and 30% on sales, said EU pricing was a matter for its Italian partner, although the Dutch firm does plan to discuss Glybera pricing during an investor meeting in New York next month.

With only 150 to 200 patients likely to be eligible for Glybera across Europe, the impact on healthcare budgets will be small, even at a very high price – but this case will be watched closely as a benchmark for future gene therapies.

UniQure also has plans to seek approval for Glybera in the United States, which it hopes to get in 2018.

Although there is already a gene therapy for cancer on the market in China, that has not been rolled out to other countries, making Glybera a first for the west.

Proponents of the gene-fixing technology insist it stacks up as a cost-effective treatment, despite the high cost, as it could permanently cure many patients.

In the case of Glybera, Chiesi said the annualised cost was no more than that charged for some expensive enzyme replacement therapies.

UniQure is also working on gene treatments for haemophilia and has an early-stage project in heart failure.

Assuming trials are successful, analysts expect gene medicines treating more common conditions to cost less, as manufacturers should be able to recoup their research and development investment from a larger patient group.

Rivals in the gene therapy market include privately-owned Spark Therapeutics, which has an eye drug in late-stage clinical tests, and Bluebird Bio, which is working on drugs for neurological and blood disorders.

Bluebird Bio and UniQure both staged successful floats on the Nasdaq market in the past 18 months, reflecting growing investor interest in the field. Among major pharmaceutical companies, Bayer struck a gene therapy deal with Dimension Therapeutics in June, while Novartis recently established a new cell and gene therapies unit, and Sanofi has a long-standing tie-up with Oxford BioMedic.

courtesy : http://www.theguardian.com/

ഫ്രാങ്ക്ഫര്‍ട്ട്: കാല്‍നൂറ്റാണ്ട് കാലത്തെ പരീക്ഷണങ്ങള്‍ക്കും തിരിച്ചടികള്‍ക്കുമൊടുവില്‍ ജനിതക ചികിത്സാ മരുന്ന് വിപണിയിലെത്തി. വിലയിലും റൊക്കോഡ് കുറിച്ചാണ് പുത്തന്‍ സാങ്കേതികവിദ്യാ മരുന്നിന്റെ വരവ്.

ഡച്ച് കമ്പനി യുണിക്യൂര്‍ പുറത്തിറക്കിയ ഗ്ലിബറ എന്ന പേരിലുള്ള മരുന്നിന് 14 ലക്ഷം ഡോളറാണ്(ഏകദേശം എട്ടരക്കോടി രൂപ) വില. അത്യപൂര്‍വ ജനിതകരോഗം ലിപ്പൊപ്രോട്ടീന്‍ ലിപാസി ഡഫിഷ്യന്‍സി(എല്‍.പി.എല്‍.ഡി.) ചികിത്സയ്ക്കുള്ളതാണ് മരുന്ന്. രക്തത്തിലെ കൊഴുപ്പ് വിഘടിപ്പിക്കതെ കട്ടപിടിക്കുന്ന ജനിതക രോഗമാണിത്. രോഗിക്ക് 42 കുത്തിവെപ്പുകളാണ് വേണ്ടത്. ഇതിനാണ് ഇത്രയും ഭീമമായ തുകവേണ്ടത്.

യൂറോപ്പിലാകെ 150- 200 എല്‍.പി.എല്‍.ഡി. രോഗികളാണുള്ളത്. അടുത്തവര്‍ഷം ആദ്യം ഗ്ലിബറ ഉപയോഗിച്ചുള്ള ചികിത്സതുടങ്ങാനാവുമെന്നാണ് പ്രതീക്ഷ. തകരാറുള്ള ശരീരകോശങ്ങളിലേക്ക് ശരിയായജീനുകള്‍ കടത്തിവിടുന്നതാണ് ജനിതക ചികിത്സാരീതി. വര്‍ഷങ്ങളായി ഗവേഷണം നടക്കുന്ന മേഖലയാണിത്. 

courtesy : http://www.mathrubhumi.com/