Pharming seeks marketing authorization for recombinant human C1 inhibitor in Europe

By E. Janene Geiss

Philadelphia, July 21 - Pharming Group NV said Friday that it has submitted the Marketing Authorization Application for recombinant human C1 inhibitor (rhC1INH) to the European Medicines Agency for the treatment of acute attacks of hereditary angioedema.

Pharming said its application is supported by clinical and pharmaceutical data on rhC1INH as required by the EMEA, according to a company news release.

In clinical studies, all hereditary angioedema patients treated with rhC1INH demonstrate a rapid time to beginning of relief (typically less than two hours) and time to minimal symptoms (typically less than 12 hours), officials said.

The data from preclinical and clinical studies reinforce rhC1INH's safety and effectiveness with rapid and sustained relief for patients, the company said.

The rhC1INH product has already received Orphan Medicinal Product Designation for hereditary angioedema in Europe, which provides 10 years of market exclusivity for an approved recombinant human C1 inhibitor product.

The company said it expects to provide further information on rhC1INH at an appropriate time and in a manner that reflects the ongoing interactions with EMEA for approval of the product.

Pharming said its rhC1INH could represent the first new therapy for hereditary angioedema patients in more than 30 years if approved. The only approved product for the treatment of hereditary angioedema attacks is plasma derived C1 inhibitor, which is available in a limited number of European countries.

Hereditary angioedema is a genetic disorder caused by a shortage of C1 inhibitor activity.

About one in 30,000 individuals suffers from hereditary angioedema and has an average of seven acute attacks per year. Hereditary angioedema attacks that are untreated usually last up to five days. The disease is characterized by acute attacks of painful swelling of soft tissues, 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.

Pharming said it has secured the large scale commercial production of rhC1INH through its collaboration with the Akzo Nobel subsidiary Diosynth BV.

Based on the standard schedule for accepted applications using the centralized procedure, Pharming said it anticipates responding to questions from EMEA and coordinating inspections in 2006.

Pharming is a Leiden, The Netherlands, biopharmaceutical company.

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