PHARMING WINS ORPHAN PRODUCT GRANT FOR HAE DRUG

Pharming announced that it has received a grant from the FDA's Office of Orphan Products Development (OPD) for the clinical development of recombinant human C1 inhibitor (rhC1INH or Rhucin) for treatment of attacks of hereditary angioedema (HAE).

The OPD grant program provides funding for clinical development of products for rare diseases with limited or no treatment options. The maximum one-year grant for clinical development of Rhucin amounts to $344,861. Pharming is eligible to receive additional funding for Rhucin development from the OPD after this period if appropriate.

Rhucin has orphan drug designation for the treatment of attacks of HAE. Orphan drug designation is reserved for promising new therapies being developed to treat diseases that affect fewer than 200,000 people in the United States. This designation provides an accelerated review process, certain tax advantages, eligibility for grants for clinical studies and a seven-year period of market exclusivity upon product approval.

In the United States, the rhC1INH product is being studied in a randomized, placebo-controlled, double-blind clinical trial for the treatment of patients with acute attacks of HAE. There is currently no approved therapy available in the U.S. for the treatment of acute HAE attacks.

HAE is a human genetic disorder caused by a shortage of C1 inhibitor activity. The disease is characterized by acute attacks of painful and in some cases fatal swelling of several soft tissues.