PARI Granted Orphan Drug Designation for Inhaled Cyclosporine – Potential New Therapy for Lung Transplant Patients

MONTEREY, CALIFORNIA, August 23, 2004 – On July 29, PARI GmbH was granted Orphan Medicinal Product designation for inhaled cyclosporine in the European Union. PARI intends to develop inhaled cyclosporine to minimize the risk of graft rejection in lung transplants. This is the first drug the company will privately develop in conjunction with its new eFlow® aerosol delivery platform, an electronic, portable inhalation device that can be customized based on medication and treatment needs.

While lung transplants are a common treatment for patients suffering from incurable lung disease and related diseases causing degradation of the lungs (such as emphysema and cystic fibrosis), only about 45% of transplant patients survive more than five years.

Currently, cyclosporine is used as a standard oral therapy to systemically create high doses of medication in the body and a desired concentration in the lungs. PARI believes that in the form of an inhaled treatment, cyclosporine can reach desired concentration levels in the lung without the high systemic penetration. This provides the potential for reduced side effects that today include kidney and brain damage and vulnerability to infection.

Historically, cyclosporine has poor solubility in water. This has created varied drug concentrations in lung tissue when prepared for routes differing from oral dosages. PARI has found a way to combat these issues with eFlow.

PARI’s eFlow, an electronic, portable nebulizer, enables a very efficient aerosolization of liquid medications via a vibrating, perforated membrane. Compared to other nebulizer systems, eFlow can produce aerosols with a high density of active ingredients, a defined droplet size, and a high proportion of respiratable droplets in a shorter amount of time. Its silent mode of operation, small size (it fits in the palm of your hand), light weight and possibility of battery use make this device extremely user-friendly.

In clinical studies, PARI’s scientists aim to prove the safety and efficacy of cyclosporine when given as inhalation therapy with eFlow. Based on laboratory tests with artificial lung models, PARI believes that the desired therapeutic effect can be achieved with notably reduced drug doses when given via inhalation, compared to oral therapies. The result could be improved quality of life and survival chances for lung transplant patients.

PARI sought Orphan Medicinal Product designation for inhaled cyclosporine to fulfill an unmet medical need. With the Orphan Medicinal Product designation, PARI receives market exclusivity for 10 years and has access to European Union incentives that include protocol assistance, access to the centralized procedure of the European Agency for the Evaluation of Medicinal Products, potential fee reductions and possible EU funded research. To qualify for orphan status, medicinal products must diagnose, prevent, or treat life-threatening or very serious conditions that are rare and affect not more than 5 in 10,000 persons in the EU.

PARI’s decision to seek market approval for inhaled cyclosporine is a new direction for the company. For many years, PARI has worked closely with pharmaceutical companies to bring inhaled products to market and has established competence in the development and testing of drugs.

”We are very interested in developing tailor-made products for nasal and pulmonary application that seek to improve medication treatment and quality of life for patients,” said Dr. Manfred Keller, Director of PARI Business Unit Pharma. “In cooperation with lung transplant specialists, we will create a clinical development program for the cyclosporine project and begin the pharmacokinetic and clinical studies as soon as possible.”

PARI will also continue to work closely with pharmaceutical companies to develop other customized drug-device therapies with eFlow. Because eFlow provides the potential for significantly reduced medication doses and treatment times, PARI is in the unique position of helping pharmaceutical companies develop innovative new medication formulations with customized drug-device matching. The potential for creating super-generics, or new uses for approved active ingredients, is also greater with eFlow due to its innovative technology platform.

About PARI
PARI is a leading worldwide manufacturer of fast and efficient aerosol delivery products for patients with asthma, chronic lung disease, and cystic fibrosis. PARI's primary focus is to provide patients with innovative products and services that help control disease. PARI is headquartered in Starnberg, Germany and has offices in the United States, Japan, United Kingdom, and Canada. Additional information on eFlow is available by contacting Kirsten Ayars at 805-898-0567 or Geoff Hunziker at 408-892-2097.

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