PARI Granted Orphan Drug Designation for Inhaled Cyclosporine Potential for a New Treatment of Rejection Reactions Following Lung Transplants

STARNBERG, Germany 27. 08. 2004 (PROTEXT) - For over twentyyears, lung transplants have been a commonly accepted treatmentmethod throughout the world for patients with serious, incurablelung diseases. Until now however, acute and chronic rejectionreactions to the implanted organ have lowered the patientschances of survival: only 45 percent of patients survive forlonger than five years.

Researchers and developers at PARI GmbH - specialists foreffective inhalation - consider the administration of inhaledCyclosporine to be much superior to conventional treatments usedin the prevention and treatment of rejection reactions followinglung transplants. On June 16, 2004, the European licensingauthority granted PARI ODD (Orphan Drug Designation) status forthe inhaled Cyclosporine therapy hereby giving PARI the economicadvantage of having market exclusivity for Cyclosporine for aperiod of 10-years. The licensing authority helps promote thediscovery, development and marketing of medications for rarediseases.

Cyclosporine, a calcineurine immunosuppressor, is amedication used frequently following transplant operations forthe suppression of the bodys natural rejection of a foreignorgan. PARI scientists are now preparing clinical studies todetermine the necessary dosage of an innovative aqueousCyclosporine formulation when administered with the new, highlyefficient PARIeFlowŽ membrane nebulizer. In clinical trials, anAmerican group of researchers has already determined thatinhaled Cyclosporine therapy has advantages over the conventionalorally administered therapy because the drug reaches the criticalarea, the transplanted lung, directly.

Based on results from research with artificial lung models,the PARI scientists expect that, when administered with theeFlowŽ, the desired therapeutic effect can be achieved withsignificantly lower doses of inhaled Cyclosporine compared tooral therapies while simultaneously minimizing side effects.This should significantly improve the patients chances ofsurvival and quality of life.

The current therapy with orally administered Cyclosporinerequires high doses of medication in order to produce the desiredconcentration in the lung, as the active ingredient isdistributed in the entire body. This causes a great number ofdosis-dependent side effects, notably kidney and brain damage aswell as an increased vulnerability to infections. Due to the poorsolubility of Cyclosporine in water, the biological availabilityof the active ingredient and consequently the concentration ofCyclosporine in the lungs vary from patient to patient.

The oscillating, perforated membrane of the new electronicPARI-eFlowŽ nebulizer supports an efficient way of atomizingliquid medications. Compared with other nebulizers, eFlowŽ isable to deliver aerosols with a predefined droplet size and ahigh proportion of active ingredient reaching the lungs withinthe shortest possible time. Soundless operation, smalldimensions, low weight, as well as the possibility of batteryoperation, make this device extremely user-friendly.

Receiving ODD status for inhaled Cyclosporine by the Europeanlicensing authority confirms the fact that the Pharma businessunit constitutes an important element in the PARI companystructure and improves the companys prospects for the future.The Pharma drug development and testing expertise enables PARI toexpand its original inhalation device business to includeinhalable medication and thus become a system provider. In thisway we are able to develop customized products for nasal andpulmonary applications that improve drug therapy and the qualityof life of our patients, states Dr. Manfred Keller, Director ofthe PARI Business Unit Pharma. In collaboration with transplantcenters we will design a clinical development program for theCyclosporine project so we can begin the necessarypharmacokinetic and clinical studies as soon as possible.

Orphan drugs are medications for the treatment of rarediseases. In Europe, the licensing authority for the developmentof such medications has been granting orphan drug status forselected medications since December 1999 providing incentives topharmaceutical companies through shortened approval times andmarket exclusivity for a period of ten years. This promotes theresearch, development and launching of drugs for the treatment ofrare diseases. In the USA, such incentives exist since 1983, inJapan since 1993.

Internet: http://www.presseportal.de

Contact: PARI Aerosol Research Institute Dr. Johannes-Christoph Stein Steinerstrasse 15k D-81369 Munich, Germany Tel. +49-89-74-28-46-69 Fax +49-89-74-28-46-50 e-mail:j_stein@pari.de

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