Alexion Pharmaceuticals, Inc. ALXN announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion regarding the update of the therapeutic indication for Soliris. Soliris is approved for the treatment of two severe, ultra-rare and life-threatening disorders – paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.
Upon the EC’s approval, the updated information related to the use of Soliris for the treatment of PNH and aHUS will be available in the Summary of Product Characteristics, which will be published in the revised European Public Assessment Report.
According to the CHMP’s opinion, patients suffering from PNH and at high risk of morbidity or premature mortality can benefit from Soliris, regardless of their history of transfusion. The opinion was based on the efficacy and safety data from an observational, non-interventional international PNH Registry, which determined that patients without any history of transfusion showed significant reduction in hemolysis and related symptoms including fatigue, when treated with Soliris.
As far as aHUS is concerned, the CHMP is in favor of adding the new efficacy data to the EU product label, which specifies that long-term treatment with Soliris helped an increased percentage of patients to achieve clinically meaningful improvements. Notably, with the continuation of treatment beyond 26 weeks, patients achieved hematologic normalization.
The CHMP’s opinion was based on additional data from ongoing long-term follow-up studies on patients, who were previously evaluated in studies for aHUS. If approved by the EC, the Section 4.4 of the EU label will include new information regarding the risks associated with the discontinuation of treatment with Soliris in aHUS patients.
We believe that EU label updates for Soliris should strengthen the drug’s position in the market. It may even increase demand for Soliris by PNH patients who have not undergone transfusion as well as aHUS patients for the continuation of their treatment regimes, thereby improving the drug’s performance as well as Alexion’s top line.
Meanwhile, Alexion is working for label expansion of Soliris. The company is evaluating Soliris for several additional indications including delayed kidney transplant graft function, neuromyelitis optica and myasthenia gravis. We expect investor focus to remain on updates on Soliris development.
Alexion carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are ANI Pharmaceuticals, Inc. ANIP, Cytokinetics, Incorporated CYTK and Isis Pharmaceuticals, Inc. ISIS. All these carry a Zacks Rank #1 (Strong Buy).
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