Medical Need announces that the product GranuPAS® has been granted EU marketing authorization by the European Commission on April 7, 2014. The product contains para-aminosalicylic acid (PAS) in a gastro resistant coated granular formulation. PAS is used in the treatment of multi-drug resistant tuberculosis (MDR-TB) in combination with other anti-tuberculosis agents. The gastro-resistant formulation prevents formation of a liver toxic metabolite in the acidic environment of the gut and reduces gastrointestinal problems, which may be associated with the substance in its uncoated form.
The product has a marketing authorization in the US under the trade name Paser®, but has up until now only been available in Europe on a named patient basis. Following the decision by the European Commission, the product is now approved as an orphan drug within the EU, reflecting the significant benefit of the product in this severe condition. As a result, the product is protected by orphan market exclusivity for a period of ten years.
The product is part of Medical Needs partnership with the French company Lucane Pharma, which will be the EU marketing authorization holder for the product. Under the collaboration, Medical Need holds the exclusive rights to the product in Czech Republic, Denmark, Estonia, Finland, Hungary, Iceland, Latvia, Lithuania, Norway, Poland, Slovakia, Slovenia and Sweden, and will be responsible for the distribution, marketing and sale of the product in this territory.
“Multi-drug resistant tuberculosis is a major threat to public health globally, and is a significant and growing problem. GranuPAS® is one of the important pharmaceuticals used to cure this severe disease, and to prevent further development of resistance among tuberculosis bacteria and spread of tuberculosis in the society”, said Kennet Rooth, COO of Medical Need, and continued: “We are very happy about this decision by the European Commission, which allows for easier and better access to the product to the MDR-TB patients across Europe.”
About Multi-Drug Resistant Tuberculosis (MDR-TB)
Tuberculosis is a severe and potentially lethal bacterial infection caused by Mycobacterium Tuberculosis. It primarily affects the lungs, but may also affect other parts of the body. While Tuberculosis was previously decreasing in the western world over the course of the last century, it has during the past decades started to increase again. Globally, tuberculosis is a significant problem and is the type of bacterial infection responsible for the highest number of deaths in adults. Individuals with impaired immune system are particularly susceptible to infection, why there is a large co-morbidity in patients affected by HIV. Globally it is estimated that approximately 8 million people will develop active disease on a yearly basis and 1.7 million will die from the disease every year.
Multi-drug resistant tuberculosis (MDR-TB) is defined as infection by a strain of mycobacterium tuberculosis, resistant to at least two of the first-line anti-tuberculosis medications. It complicates and limits the possibility to cure the affected individuals and to prevent spread of the disease in the society. Like for other bacteria, resistance develops when the levels of antibiotics in the affected patients are insufficient to kill all the bacteria, which may be caused by poor compliance to the treatment regimen for example due to lack of medication or funding. In recent years, bacterial strains with very broad resistance pattern to many antibiotics (extensively drug resistant (XDR-TB)) have started to emerge and are of particular concern. XDR-TB currently accounts for approximately 10% of MDR-TB world-wide.
MDR-TB should be treated with a combination of several anti-tuberculosis medications, often up to five different substances and usually for a period of up to two years, in order to secure complete eradication of the bacteria and prevent further development of resistance. Left untreated, MDR-TB has a mortality rate of as high as 80%. MDR-TB is considered a major threat to global public health by the WHO, which currently estimates approximately 0.5 million new cases of MDR-TB are reported each year, the vast majority of which in the developing world. Over the past decade, MDR-TB has become an increasing problem also in the EU and the Nordic countries, partly linked to immigration.
GranuPAS is approved for the treatment of MDR-TB, as part of an appropriate combination regimen, in adults and paediatric patients from 28 days of age and older, when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.The product contains para-aminosalicylic acid formulated in gastro resistant coated granules. The gastro resistant formulation prevents formation of a hepatotoxic substance; meta-aminophenol, which is otherwise formed upon contact of para-amino salicylic acid with the acid environment in the ventricle. The product is usually dosed three times per day and is provided in convenient sachets, which contain a typical dose for adults. For children or patients requiring different dosage, a validated dosing spoon has been developed to facilitate and secure administration of the correct dose.
The product is already approved in the US under the trade name Paser®, but has up until now only been available in Europe on a named patient basis under special license application by a specialist prescriber to the national regulatory authorities. The product holds an orphan drug designation within the EU, reflecting the significant benefit of the product in this severe condition. As a result of the marketing authorization, the product is now protected by orphan market exclusivity for a period of ten years.
Medical Need holds the exclusive rights to the product in Czech Republic, Denmark, Estonia, Finland, Hungary, Iceland, Latvia, Lithuania, Norway, Poland, Slovakia, Slovenia and Sweden, and will be responsible for the distribution, marketing and sale of the product in this territory.
About Orphan Drug Designation
The EU orphan drug legislation was adopted in the year 2000 with the goal of stimulating development of pharmaceuticals for rare conditions with unmet medical needs, so called orphan drugs. A rare condition is defined as a condition affecting less than 5 out of 10 000 individuals across the EU. Under the legislation, companies developing such pharmaceuticals may apply for an orphan drug designation of their product, which makes it eligible for several incentives, including free assistance, fee reductions and waivers throughout the development and, following marketing authorization, ten years market exclusivity.
About Lucane Pharma
Lucane Pharma is a privately owned pharmaceutical company, Paris based, set up end 2009. Lucane Pharma develops and or inlicenses, registers and commercializes medicinal products for rare conditions. Since its inception, Lucane Pharma has already obtained the EU registration for its first developmenet product Pheburane. The EU registration of GranuPAS (April 2014) is the second one granted to Lucane Pharma. Several other developments are currently ongoing in various areas, including Metabolic Disorders and Tuberculosis.