FDA approves Investigational New Drug application for Prodarsan® in
Cockayne Syndrome patients
(Thomson Reuters ONE) - Leiden, The Netherlands, August 14, 2009. Biotech company PharmingGroup NV ("Pharming" or "the Company") (NYSE Euronext: PHARM)announced today that its wholly-owned subsidiary, DNage, has had itsInvestigational New Drug (IND) application for Prodarsan® accepted bythe US Food and Drug Administration (FDA), allowing DNage to initiatea clinical trial in children suffering from Cockayne Syndrome (CS).In this short clinical study, which is expected to be conducted inthe fourth quarter of 2009, the pharmacokinetics (PK) of single andmultiple escalating doses of Prodarsan® will be evaluated in a smallnumber of paediatric patients with CS. The individual components ofProdarsan® are already known to the FDA and during this study datawill be collected to make a bridge to existing data on the individualcomponents of Prodarsan®. This will facilitate the futureregistration of Prodarsan®. In addition to data collected in previousstudies, the safety and tolerability of the product will also beinvestigated in this study. The Principal Investigator is Dr. EdwardG. Neilan, Staff Physician at the Children's Hospital Boston/HarvardMedical School, a world-renowned clinician and researcher in thefield of Cockayne Syndrome.Prodarsan® is a product in development to delay the progression ofCS, a premature ageing disease. Premature ageing diseases are a groupof rare diseases caused by a genetic defect leading to deficientrepair of DNA damage. Patients suffering from these diseases developmultiple 'ageing-related pathologies', normally associated with oldage, early on in their lives. Generally, these patients have astrongly reduced quality of life and reduced life expectancy.Prodarsan® is a combination of small molecules formulated as a liquidfor oral use and is believed to reduce the accumulation ofDNA-damage, the underlying biochemical cause of CS. There iscurrently no effective therapy available for this disease.In late 2008, a Phase I study of Prodarsan® in healthy humanvolunteers was successfully completed. In this study, single andmultiple escalating doses were administered to healthy volunteers toevaluate the PK, safety and tolerability of Prodarsan® in adults.Prodarsan® appeared to be safe and well tolerated in dosages that hadearlier shown to yield significant beneficial effects in animalmodels of CS. Since Prodarsan® will be used as an oral, liquidformulation the effects of food intake on absorption and eliminationof the product were also investigated. Based on these data, combinedwith data generated in the preclinical program, an oral dosing schemehas been established that will be used in the upcoming studies inpaediatric CS patients.After successful completion of the planned clinical study and theongoing toxicology testing, DNage expects to perform another clinicaltrial to evaluate the efficacy of Prodarsan® in alleviating symptomsof CS. The final design and set-up of this planned internationalstudy will be completed after discussions with regulatory agenciesand evaluating the results of the currently planned short clinicalstudy.Dr. Rein Strijker, General Manager of DNage and Chief CommercialOfficer at Pharming, commented: "Prodarsan®, as the first productbeing developed with DNage technology, is testimony to the greatpotential of this approach in treating diseases related to ageing inhumans. The positive data with Prodarsan® in preclinical and theearlier completed Phase I studies are encouraging for its furtherdevelopment. We look forward to initiating studies in CS patients andwill also continue to develop a number of other product candidatesfor the treatment of other premature ageing diseases as well asageing diseases in the elderly population. With the continuedincrease in average life-expectancy of humans in many parts of theworld, there is a great medical need for new products in the field ofdiseases associated with old age. We believe that our innovativeDNage technology can contribute to develop new approaches in thisarea."---------------------------------------------------------------------Background on Investigational New Drug (IND) ApplicationAn Investigational New Drug application is a request forauthorization from the US Food and Drug Administration to administeran investigational drug or biological product to humans consistentwith an approved protocol. Such authorization must be secured priorto administration of any new drug or biological product. The INDapplication contains information on the product's preclinical andclinical results, manufacturing data, and detailed clinical protocolsfor proposed clinical studies. An IND approval confirms that the FDAagrees that the product can be tested in humans to collectinformation pertinent to the product's safety and efficacy. TheIND-application for Prodarsan® was filed by Clinquest, DNage's agentin the USA.Background on Cockayne SyndromeCockayne Syndrome is a rare disease and is one of a collection ofpremature ageing disorders. CS is a genetic disorder that is (amongstother symptoms) characterized by growth failure, mental retardation,hearing loss, a premature aged appearance (progeria) and prematuredeath. The average lifespan of CS patients is 12.5 years and qualityof life for these patients is seriously impaired. At present, thereis neither a cure nor an effective therapy available for CS patients.Disease management consists of treating the symptoms as they ariseand providing assistive devices. In April 2009, DNage received anOrphan Drug designation from the FDA for Prodarsan®. The Orphan Drugdesignation provides for an accelerated review process, tax benefits,exemption from user fees and a seven-year period of marketexclusivity in the US after product approval.About DNage BVDNage is a wholly owned subsidiary of Pharming Group NV since itsacquisition in 2006. DNage is developing products that interfere withthe development of ageing-related pathologies. Its technologyplatform is based on the scientific work by the group of Dr.Hoeijmakers, at the Erasmus Medical Centre in Rotterdam, whoestablished a link between insufficient DNA-repair (accumulation ofDNA-damage) and the development of ageing-related diseases. DNage hasactive programs in the field of neurodegeneration, bone disease andother ageing-related pathologies. In addition to the DNage programsPharming is developing innovative products for the treatment ofgenetic disorders, specialty products for surgical indications,intermediates for various applications and nutritional products.Pharming has two products in late stage development - Rhucin® forHereditary Angioedema and human lactoferrin for use in food products.Additional information is available on the Pharming website,http://www.pharming.com and on http://www.dnage.nl.This press release contains forward looking statements that involveknown and unknown risks, uncertainties and other factors, which maycause the actual results, performance or achievements of the Companyto be materially different from the results, performance orachievements expressed or implied by these forward lookingstatements.Contact:Sijmen de Vries, Pharming Group NV, T: +31 (0)71 52 47 400Rein Strijker, Pharming Group NV, T: +31 (0)71 52 47 400http://hugin.info/132866/R/1334818/317056.pdfThis announcement was originally distributed by Hugin. The issuer is solely responsible for the content of this announcement.
Datum: 14.08.2009 - 10:31 Uhr
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