Screen Shot 2017 11 07 at 9.48.38 AMNEW HAVEN: Achillion Pharmaceuticals, Inc. [Nasdaq:ACHN]  announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ACH-4471 for the treatment of paroxysmal nocturnal hemoglobinuria [PNH]. 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 PNH, as well.

The orphan drug news comes on the heels of a decision by Johnson & Johnson [NYSE: JNJ] to discontinue a development partnership with Achillion for its Hepatitis C Drugs. The outcome of clinical trials for the Achillion drugs in combination with J&J proved effective in curing certain varieties of Hep C. The Hep C market has existing drugs that also cure the disease.

Achillion stock soared to $15.90 in 2015 on the status of the treatment and prior to news of the collaboration in 2015 which brought more than $250 million in cash into the company. Currently the company has a market capitalization of $586 million and  a $4 share price. The company lost just under $20 million in the third quarter but still has more than $350 million in cash and securities.

“We are very pleased with the receipt of orphan drug designation from the FDA, and a positive opinion by COMP for orphan status, for ACH-4471, a first-in-class, oral inhibitor of complement factor D,” said Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. “We believe that modulation of the complement alternative pathway by targeting factor D, could provide a novel and pharmacologically differentiated approach to treating diseases of the complement system, including PNH and C3G.”

The FDA Orphan Drug Designation program provides incentives for the development of potentially promising drugs to treat, diagnose or prevent orphan diseases and disorders that affect fewer than 200,000 people in the U.S.

Paroxysmal Nocturnal Hemoglobinuria has fueled the $3 billion plus annual sales at Achillion’s neighbor Alexion Pharmaceuticals [NYSE: Alxn]. ACH -4471 is orally administered versus via infusion for Alexion’s Soliris drug. In April the company began a Phase II study for PNH, the company said the study was a “proof of concept study that will assess the efficacy, safety, and pharmacokinetics of ACH-4471 in untreated patients with PNH.”

Achillion has also identified the drug as a treatment for another rare disease C3 glomerulopathy and has previously announced its intention to initiate a Phase II trial, which has not been announced yet.