Onyx Pharmaceuticals (ONXX): Impressive. The company really seems to be hitting its stride with the multiple myeloma drug Kyprolis.
The tale of two ultra-orphan drug launches: Aegerion Pharmaceuticals (AEGR) is going to do well with Juxtapid in homozygous familial hypercholesterolemia (HoFH). CEO Marc Beer's presentation was one of the best and most convincing I saw during my three days in San Francisco.
The same cannot be said for NPS Pharmaceuticals (NPSP). Gattex is an important therapy for short bowel syndrome patients but too expensive at $295,000 per year. Payers are going to have problems with Gattex reimbursement, particularly since some patients may be taking the drug for months or even a year before finding out if it's helping or not.
Teva (TEVA) embarrassed itself by filing a Citizen's Petition with the FDA against Biogen Idec's multiple sclerosis pill BG-12. If this is indicative of the business acumen of Teva's new management team, shareholders are in for a long and lousy year.The FDA granted "breakthrough therapy" status to Vertex Pharmaceuticals' (VRTX) cystic fibrosis drug Kalydeco as a monotherapy and in combination with VX-809. What does this designation mean? Vertex executives weren't quite sure but it's probably a good thing, or at least it should be. FDA has been talking up efforts to encourage and assist in the development and approval of drugs for rare, life-threatening diseases so you'd think the agency would be incentivized to make "breakthrough" status mean something important. Stay tuned. Amarin (AMRN) CEO Joe Zakrzewski said to expect the Vascepa launch at the end of January and asked investors not to expect boffo prescription numbers right out of the gate. In other words, the Vascepa launch is going to be slow. Looking ahead, Amarin faces questions about its ability to attract a Big Pharma partner for Vascepa in the expanded "Anchor" indication of high triglyceride patients. And, will FDA approve an expanded label for Vascepa without positive results from the ongoing cardiovascular outcomes trial? Given the recent failure of similar heart-safety outcomes studies -- i.e. Merck's (MRK) Tredaptive -- it's not crazy to see FDA becoming more conservative.