Ligand Pharmaceuticals Inc. (NASDAQ:LGND) announces the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Revolade® (eltrombopag), a Novartis product, for the treatment of adult patients with acquired severe aplastic anemia (SAA) who were either refractory to prior immunosuppressive therapy (IST) or heavily pretreated and are unsuitable for hematopoietic stem cell transplantation.

SAA is a blood disorder where the bone marrow fails to make enough red blood cells, white blood cells and platelets3. Two out of every one million people in Europe and North America are diagnosed with aplastic anemia per year, a portion of which are severe cases4,5. The exact cause of the disease is still unknown, but most cases of SAA are believed to be triggered by an autoimmune reaction where the body attacks blood-forming stem cells located in the bone marrow3,6. As a result, patients with SAA are at risk for life-threatening infections or bleeding6.

Treatment of SAA is focused on increasing a patient’s blood cell count. The current standard of care includes IST or hematopoietic stem cell transplantation6. Of patients treated with IST, one-quarter to one-third will not respond and 30-40% of responders will relapse, causing symptoms to return6. Approximately 40% of SAA patients who don’t respond to initial IST die from infection or bleeding within five years of their diagnosis1.

The CHMP positive opinion is based on the results of a pivotal open-label Phase 2 study (ELT112523) and two supporting Phase 2 studies (ELT116826 and ELT116643) conducted by the National Heart, Lung and Blood Institute (NHLBI) at the National Institutes of Health (NIH). The pivotal study demonstrated a hematologic response (40%) in SAA patients treated with Revolade who had an insufficient response to IST. The most common adverse reactions (=20%) in the pivotal single-arm study of 43 patients were nausea (33%), fatigue (28%), cough (23%), diarrhea (21%), and headache (21%)7.

The European Commission will review the CHMP recommendation and is expected to deliver its final decision within three months. The decision will be applicable to all 28 EU member states plus Iceland, Norway and Liechtenstein. In August of 2014, eltrombopag (marketed as Promacta® in the USA), was approved by the US Food and Drug Administration for once-daily use in patients with SAA who have had an insufficient response to IST. Eltrombopag is also approved for SAA in Canada. (Original Source)

Shares of Ligand Pharmaceuticals Inc closed yesterday at $104.65. LGND has a 1-year high of $107.71 and a 1-year low of $41.99. The stock’s 50-day moving average is $98.41 and its 200-day moving average is $77.93.

On the ratings front, Ligand has been the subject of a number of recent research reports. In a report issued on July 21, Roth Capital analyst Joseph Pantginis maintained a Buy rating on LGND, with a price target of $135, which represents a potential upside of 29.0% from where the stock is currently trading. Separately, on June 19, Cantor Fitzgerald’s Irina Rivkind Koffler reiterated a Hold rating on the stock and has a price target of $93.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Joseph Pantginis and Irina Rivkind Koffler have a total average return of 9.6% and 55.3% respectively. Pantginis has a success rate of 51.4% and is ranked #212 out of 3714 analysts, while Koffler has a success rate of 69.6% and is ranked #3.

Ligand Pharmaceuticals Incis a biopharmaceutical company with a business model that is based upon the concept of developing or acquiring royalty revenue generating assets and coupling them with a lean corporate cost structure.