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Retinopathy of Prematurity.
FDA Grants Orphan Drug Designation for Microplasmin Use in Pediatric Eye Surgery.





April 1, 2004.

Press Release.
Source: ThromboGenics Ltd.




Plans to Accelerate Clinical Evaluation.


DUBLIN, Ireland, April 1 /PRNewswire/ -- ThromboGenics Ltd. has been granted orphan drug designation by the Food & Drug Administration (FDA) for use of its compound microplasmin in pediatric eye surgery, to treat retinal disorders that cause visual impairment and blindness in children. Orphan drug status is granted for treatments intended to prevent, diagnose or treat rare, life-threatening or chronically debilitating diseases that affect up to 200,000 people in the U.S.


Microplasmin has the potential to simplify a surgical procedure called vitrectomy, which is used to treat visual conditions such as Retinopathy of Prematurity (ROP), the leading cause of blindness in infants and young children. ROP is a result of incomplete ocular development, and occurs in 16% of all premature infants. The surgical vitrectomy involves separation of the vitreous (the gel-like substance in the center of the eye) from the retina in the back of the eye. Unfortunately, the procedure is particularly challenging in pediatric patients, where the vitreous is extremely adherent, making the surgery very difficult and prone to complications. In fact, the procedure has been likened to "removing scotch tape from tissue paper".


Microplasmin has shown effectiveness in inducing posterior vitreous detachment (PVD), which is a critical part of the vitrectomy. Dr. Michael Trese, a recognized pioneer in the surgical management of ROP and co-founder of NuVue Technologies, Inc., notes, "Any agent that might facilitate or improve success in the pediatric population would be a tremendous advance for surgeons, patients and families dealing with this difficult disease."


The FDA orphan designation comes on the heels of ThromboGenics' new licensing deal with NuVue, leaders in the research and application of vitreoretinal surgery techniques. The two companies are collaborating on development of plasmin-based products, such as microplasmin, to treat serious visual disorders.


Orphan designation promotes the development of drugs to treat rare diseases or conditions by protecting them from competition for up to seven years. It also provides the benefits of a waiver of FDA user fees and tax credits of up to 50% of the qualified clinical trial expenses.


"We are pleased and encouraged to receive orphan drug designation for microplasmin," explains Professor Desire Collen, founder and CEO of ThromboGenics. "ThromboGenics has performed extensive pre-clinical work with microplasmin for intravitreal injection, including toxicology and pharmacology studies. We have also completed a successful Phase I clinical trial for systemic administration of microplasmin. It is our intention to proceed with Phase IIa clinical development later this year."


Microplasmin in Surgical Vitrectomy


The vast majority of vitrectomies are actually performed in adults -- annually, more than 500,000 worldwide and 200,000 in the U.S. In addition to its application in the setting of surgical vitrectomy for children and adults, microplasmin also may be of benefit for preventive treatment of various conditions, including diabetic retinopathy, which can lead to loss of vision. Based on evidence from several epidemiologic studies, PVD appears to protect against diabetic retinopathy progression, as well as diabetic macular edema. Microplasmin, when given as a single injection in the physician's office to induce PVD, may provide significant benefit for patients with various forms of diabetic retinopathy.




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