Who might buy Sarepta? Take your pick of Big Pharma although I'd love to see Vertex Pharmaceuticals (VRTX) make a bid. Vertex adding eteplirsen and a new focus on muscular dystrophy would be a compelling bookend to its groundbreaking work in cystic fibrosis.
Approximately 2,000 patients have been prescribed Qsymia since Vivus (VVUS - Get Report) launched the weight-loss drug one month ago, according to prescriptions tracked separately by healthcare research firms IMS Health and Wolters Kluwer.
@kevinccc asks, "What do you think about this number so far, Adam?"
Meh. Two thousand Qsymia patients in the first month doesn't sound particularly robust, even given relatively low expectations.
Anthony E. writes, "Adam, thanks for live blogging the FDA advisory panels last week. I agree with you that Aegerion Pharmaceuticals (AEGR - Get Report) came out looking better than Isis Pharmaceuticals (ISIS) but do you think both drugs will be used? What is next for both these stocks?" Last week's Aegerion and Isis FDA panels were a great lesson in how to measure the relative risks and benefits of a drug. Aegerion's lomitapide and Isis' mipomersen can both turn livers into human foie gras, but that's an acceptable risk when balanced against their respective ability to lower cholesterol levels in patients with blood that resembles the jar of schmaltz my grandmother used to keep in her refrigerator. Based on the positive panel votes, FDA will approve both lomitapide and mipomersen for treatment of patients with homozygous familial hypercholesterolemia (HoFH). The labels will be narrow and come with restrictions in order to safeguard against unsafe use in patients with less severe increases in blood cholesterol. However, I don't expect FDA to mandate genotyping to ensure that all patients have true HoFH. This means use of both drugs could bleed into heterozygous familial hypercholesterolemia (HeFH) patients. The number of HoFH/severe HeFH patients is a guessing game. The FDA estimates 300 HoFH patients in the U.S.; Aegerion says 3,000 patients (using a more liberal definition that likely captures HeFH patients as well.) Figure pricing in the $200,000 to $400,000 range, likely depending in part on the number of restrictions FDA places in the drugs' respective labels. Another wild card is duration of treatment. About one-third of patients treated with both drugs discontinue early either due to poor response or side effects. The drugs haven't been studied head to head but Aegerion's lomitapide should capture greater market share than Isis' mipomersen for reasons I discussed at length during the live blogs: A more convenient, once-daily pill, better cholesterol-lowering efficacy and less serious side effects.
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