With the SPDR S&P Biotech Index up 36% year to date, it's very evident that investment dollars are willingly flowing into the biotech sector. Keeping that in mind, let's have a look at some of the rulings, studies, and companies that made waves in the sector last week.
It was a decisive week for homozygous familial hypercholesterolemia, or HoFH, drugmakers Aegerion Pharmaceuticals (NASDAQ: AEGR) and Isis Pharmaceuticals (IONS -0.66%) as they each went before the FDA panel with their respective drugs. The panel results demonstrated a clear bifurcation, with Aegerion coming out the winner.
By a vote of 13-2, the FDA panel voted to recommend the approval of Aegerion's lomitapide to treat the high LDL-cholesterol disease, although concerns about the drug's effect on the liver and in lower target populations still echoed throughout the panel. Ultimately, the drug may require a special risk evaluation and mitigation strategy so as to ensure patient safety and train physicians how to properly disseminate the drug and track patient health. Either way you cut it, lomitapide appears on its way to approval.
Isis' drug, Kynamro, which is being developed in collaboration with Sanofi (SNY 1.94%), wasn't nearly as lucky. In addition to the liver function concerns that worried the panel with Aegerion's lomitapide, FDA panel members expressed concern about cancerous growths that occurred in 3.1% of all tested patients. Although the panel's vote was still in favor of approval by a tally of 9-6, it wasn't a convincing meeting for Isis, and the possibility of further testing and delays have Isis shares grounded .
NPS Pharmaceuticals (NASDAQ: NPSP) didn't have a particularly good week despite news from the FDA panel that it unanimously is recommending the approval of Gattex, the company's short bowel syndrome drug. Although nothing is guaranteed in the world of biotechs, a unanimous approval from the FDA panel when compounded with the largely unmet needs of SBS patients is likely to lead to Gattex's approval by the FDA in its PDUFA on or before Dec. 30.
Microcap Aeterna Zentaris (AEZS 0.17%) rounded out the week by reporting the final data on its late-stage adult human growth hormone, or ADHD, diagnostic test, AEZS-130. An orally activated ghrelin agonist, AEZS-130 demonstrated far fewer adverse events (i.e., side effects) in studied patients and was ultimately preferred by a vast majority of them over the currently available ADHD test. The drug has been given orphan drug status, and Aeterna does own the rights to it worldwid. However, we'll need to wait on an FDA decision until next year as it was recently denied fast-track status .
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