Achillion Pharmaceuticals, Inc. (NASDAQ: ACHN) gained ground, after the company disclosed that ACH-4471 has received positive opinion for orphan drug designation in the European Union for the treatment of C3 glomerulopathy.
The company, based out of New Haven, Connecticut, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on ACH-4471 for orphan status in the European Union (EU) for the treatment of C3 glomerulopathy (C3G).
According to Achillon CEO Milind Deshpande, "We are very pleased that ACH-4471, a first-in-class, oral inhibitor of complement factor D, has received a positive orphan status opinion for the treatment of C3G.
"At Achillion, we continually work to enhance the awareness of C3G, a rare renal disorder consisting of dense deposit disease (C3G) and C3 glomerulonephritis (C3GN) for which there are no approved therapies."
C3G is a devastating disease affecting the kidney for which there is no approved therapy. C3G affects persons of all ages with men and women equally affected. There are estimated to be approximately 4,000 C3G patients in the United States, more than 4,000 in Europe, and greater than 1,000 patients with this disease in Japan.
C3G describes a rare renal disease characterized by the presence of C3 protein fragments in the filtering units (glomeruli) of the kidney.
Shares in Achillon moved into noon hour ET on Monday ahead 35 cents, or 12%, from Friday’s close, to $3.31, within a 52-week trading range of $2.58 to $5.66.
