Pharming Group NV announced that the US Food and Drug Administration (FDA) has approved the company's investigational new drug (IND) application for recombinant human C1 inhibitor (rhC1INH). Based on this approval, the company has expanded the clinical development of rhC1INH for the treatment of hereditary angioedema to the United States.

Pharming will assess the safety and efficacy of rhC1INH to treat acute attacks of hereditary angioedema (HAE) in its US clinical trials. The clinical protocol involves a randomized, double blind, placebo controlled trial for rhC1INH in HAE patients to be conducted at multiple centers throughout the US. The company will test rhC1INH in patients with acute attacks of HAE with two doses of rhC1INH.

"The use of rhC1INH as a replacement therapy may offer significant treatment benefits for HAE patients as the product addresses the underlying genetic basis of the disease, namely a shortage of C1 inhibitor," said Dr. Jan Nuijens, senior director of Clinical Development at Pharming. "We look forward to working with investigators and patients in the United States participating in our clinical trials with rhC1INH."

In clinical studies in Europe, HAE patients treated with rhC1INH show a significant decrease in time to beginning of relief and time to complete resolution with no adverse effects reported to date.

"We are excited to receive the FDA's approval of the rhC1INH IND and to expand our clinical trials to the United States, which represents the largest single market in the world," said Dr. Francis Pinto, CEO of Pharming. "The approval of the IND underscores the potential value of rhC1INH for the treatment of HAE."

Pharming is currently conducting a randomized, double blind, placebo controlled Phase III clinical trial for rhC1INH across Europe and remains on track to file for approval of the product with the European Agency for the Evaluation of Medical Products (EMEA) in 2005.

An IND application is a request for authorization from the US Food and Drug Administration (FDA) to administer an investigational drug or biological product to humans consistent with an approved protocol. Such authorization must be secured prior to interstate shipment and administration of any new drug or biological product. The IND application contains information on the product's preclinical and clinical results, manufacturing data, and detailed clinical protocols for proposed clinical studies. An IND approval confirms that the FDA agrees that the product can be tested in humans to collect information pertinent to the products safety and efficacy.

HAE is a genetic disorder caused by C1 inhibitor deficiency. In the West, approximately 1 in 30,000 individual suffers from HAE and has an average of 7 acute attacks per year. 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 may be seriously impaired. HAE attacks that are untreated typically last up to 5 days. Current treatment of HAE consists of prophylaxis and management of acute attacks. Attacks of angioedema can be effectively treated with intravenous administration of C1 inhibitor purified from human blood. However, C1 inhibitor preparations obtained from human blood have only been approved for use in some European countries. Although blood derived product has been shown to be effective for HAE, treatment with such plasma-derived preparations has potential drawbacks in terms of safety and product supply.