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FDA Grants Orphan Drug Status to Monarsen, Potential Treatment for Myasthenia Gravis

HERZLIA, ISRAEL -- December 3, 2003 -- Ester Neurosciences announced today that the U.S. Food and Drug Administration has granted the company Orphan Drug Designation status for Monarsen, (formerly known as EN101), for the treatment of myasthenia gravis (MG).

Orphan Drug Designation status gives Ester, upon marketing approval, the exclusive right to market a drug of this kind for MG in the US for seven years. In addition to marketing exclusivity, the advantages of the designation include eligibility for research grants to conduct clinical trials, certain tax benefits, and an exemption from certain user fees at the time of submission for marketing approval of a new drug application. A similar Orphan Drug application has been made to European regulatory authorities.

"Obtaining orphan drug designation marks an important step in our regulatory strategy for Monarsen," said Dr. Eli Hazum, CEO of Ester Neurosciences.

"Current MG treatments which include anti-cholinesterases, steroids and immunosuppressants, offer limited efficacy and often cause unpleasant and sometimes dangerous side effects. Monarsen offers the prospect of an efficacious and safe product that can address a very large market," added Dr. Hazum.

The results of a successful Phase Ib trial for Monarsen were presented at a special Late Breaking Science session of the National Academy of Neurology earlier this year.

The breakthrough study was the first demonstration of the safe and effective use of an orally-administered anti-sense therapy for a neurological disease.

This study, where sixteen patients received oral liquid Monarsen, demonstrated significant improvement in MG symptom severity, with no cholinergic effects, nor significant adverse events. Fourteen out of sixteen patients had better scores on the Quantitative Myasthenia Gravis (QMG) scale on the last day of dosing as compared to the initial baseline. Improvement of total QMG score for these days ranged from 27.8% to 53.4% (p Monarsen is based on pioneering research carried out by Prof. Hermona Soreq of the Hebrew University, who serves as Ester's Chief Scientific Advisor. Monarsen offers a novel mechanism of action for the control of an isoform of the acetylcholinesterase enzyme that is believed to play a key role in the onset and progression of MG.

Myasthenia gravis is a chronic and debilitating autoimmune disease in which the body's immune system attacks acetylcholine receptors at the neuromuscular junction, interfering with normal muscular function. MG is characterized by general muscle weakness and excessive fatigue. In severe cases the disease can involve the respiratory muscles, causing potentially life-threatening respiratory failure.

Source: Ester Neurosciences

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