From rxpgnews.com
Recombinant human C1 Inhibitor Trial Shows Positive Results in Treating Hereditary Angioedema
By Pharming Group N.V.
May 2, 2005, 12:22
Pharming Group N.V. ("Pharming" or "the Company") (Euronext:PHARM) (PHAR.AS) announced today clinical results from Phase II/III clinical studies with recombinant human C1 inhibitor ("rhC1INH") for the treatment of hereditary angioedema ("HAE"). The positive results were reported from the open label clinical studies being conducted in five European countries at the 4th C1-INH Deficiency Workshop in Budapest, Hungary.
Patients treated with rhC1INH showed rapid time to beginning of relief (median time: 30 min) and time to resolution (median time: 4hrs). Time to beginning of relief was between 15 min to 2 hrs and time to resolution was between 1 hr to 24 hrs in treated patients.
HAE attacks which are untreated typically last up to 5 days and require medical care in a physician's office and/or hospital. These rapid results with rhC1INH include 12 treated attacks in 10 HAE patients from the on-going open label Phase II/III clinical studies. The site of attacks treated in the HAE patients were facial (25%), peripheral (17%), uro-genital (8%) and abdominal (50%). None of the patients treated with rhC1INH experienced any drug-related adverse events nor did any patient experience a relapse of the initial HAE attack.
"The results from the open label Phase II/III clinical study provide further support for the use of rhC1INH as a potential treatment for hereditary angioedema," said Dr. Jan Nuijens, Senior Director of Clinical Development at Pharming. "We are pleased by the promising safety data of rhC1INH and its ability to provide rapid relief to HAE patients."
Including previous studies, over 35 infusions of the rhC1INH have been administered to HAE patients. Over 10 patients have been infused with rhC1INH at multiple times. None of the patients infused with rhC1INH have experienced any drug related adverse events or clinically relevant immune responses.
Pharming is conducting a randomized placebo-controlled double-blind clinical trial for rhC1INH across Europe and North America. The Company will continue to treat patients in the above open label clinical study to collect safety and efficacy data on rhC1INH for its regulatory filings. In addition, Pharming has also initiated pre-clinical studies with rhC1INH for specific cardiovascular and inflammatory diseases to explore new indications beyond HAE.
Background on Hereditary Angioedema
Hereditary angioedema is a genetic disorder caused by a deficiency of the human C1 inhibitor protein. Approximately 1 in 30,000 individuals suffer from HAE and have an average of 7 acute attacks per year. HAE attacks that are untreated typically last up to 5 days.
The disease is characterized by acute attacks of swelling of soft tissues (edema), including regions of the skin, the intestine, and the mouth and throat. If the soft tissue of the throat is involved, an attack of angioedema can be fatal. In addition to the life-threatening nature of the disease, quality of life for individuals with the disease may be seriously impaired.
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