Alexion's (ALXN) Rare Disorder Drug Gets EU Orphan Status

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced that the European Commission has granted orphan drug designation to ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

The European Commission usually grants orphan medicinal product status in order to provide incentives to candidates that are being developed to treat, prevent or diagnose diseases or conditions affecting no more than 5 in 10,000 people in the EU. In addition to providing Alexion with certain benefits and incentives, this status will also allow ALXN1210 to enjoy a period of market exclusivity in the EU, upon approval.

We note that ALXN1210 is a longer-acting C5 antibody being developed by Alexion for the treatment of patients with PNH, a life-threatening and ultra-rare genetic blood disorder.

Currently, Alexion is evaluating ALXN1210 in a dose-escalating, phase I/II study and an open-label, multi-dose phase II study in patients with PNH. Preliminary results from ongoing studies on ALXN1210 have shown rapid, complete, and sustained reduction of free C5 activity and a terminal half-life of more than 30 days than that of Soliris, which may facilitate a monthly or longer dosing interval.

The company also intends to evaluate ALXN1210 for the treatment of patients with atypical hemolytic uremic syndrome (aHUS) this year.

We note that Soliris is Alexion’s key growth driver, which is approved for the treatment of both PNH and aHUS. Alexion is also developing Soliris for several additional indications including generalized myasthenia gravis and delayed kidney transplant graft function among others.

Alexion is a Zacks Rank #4 (Sell) stock. Some better-ranked stocks in the health care sector include ANI Pharmaceuticals, Inc. (NASDAQ:ANIP) , Retrophin, Inc. (NASDAQ:RTRX) and Bristol-Myers Squibb Company (NYSE:BMY) . All three stocks sport a Zacks Rank #1 (Strong Buy).