Company fortunes often ride on U.S. Food and Drug Administration decisions. Just this past week, shares of GTx (GTXI) plunged almost 50 percent after the biotech received notice from the agency that it wanted additional data before it would consider approving the company’s osteoporosis drug toremifene, which is being developed to reduce fractures in men with prostate cancer receiving androgen deprivation therapy. The FDA decision was no surprise to investors, however, as there were concerns about the  compound’s safety and efficacy in the clinic. Some investors had bet against it, shorting the stock throughout the summer and fall.  

 

Gloucester Pharmaceuticals, on the other had, is riding high. The U.S. Food and Drug Administration has given the privately-held biotech the go-ahead to sell its cutaneous T-cell lymphoma or CTCL drug, Istodax, as a treatment for patients who have received at least one prior systemic therapy. Istodax is a histone deacetylase or HDAC inhibitor and is expected to be commercially available in January 2010. This approval came a few days ahead of the expected date and was no surprise considering that an advisory panel to the FDA had given a unanimous recommendation to approve the treatment for the rare skin malignancy in September.

 

Four other companies expect to hear from the FDA in the upcoming week on whether or not they can begin to market their treatments. First up is Xenoport (XNPT) which is due to get a verdict from the FDA by the beginning of the week for its drug Solzira as a treatment for restless legs syndrome. Shares of the company have been hovering around $20 for most of the year, half their value a year ago. Positive data in September from a mid-stage trial of Solzira to treat nerve pain following shingles spiked shares up 25 percent but they have been declining since then, trading at $17.98 at the close of the week. While investors seem to lack confidence in the drug’s chances of approval, the majority of analysts expect the drug to be approved based on its efficacy and safety record.

 

Cadence Pharmaceuticals (CADX) expects to hear by November 13 whether or not it can begin to market its drug Acetavance for post operative pain and fever. JMP Securities upgraded the company’s stock in October and most people expect that the FDA will grant its approval, based on strong positive results from a late-stage trial that were reported in December 2008. Shares of the company have maintained a steady rise since then although they are trading below their high of $12.68 in midsummer. As Acetavance is Cadence’s only product at this point, an approval is imperative to the company’s health.

 

Genzyme (GENZ) has had trouble this year with the forced closure of a production facility with a resultant decline in sales of Cerezyme. Cerezyme may soon face competition from Shire’s therapy for Gaucher’s disease, which the FDA is expected to act on in early 2010. Now that production problems have been remedied, Genzyme is pinning its hopes on Lumizyme, a potential blockbuster that was originally submitted for approval as Myozyme. The FDA rejected the submission, considering the product manufactured at the new bioreactor to be substantially different biologically and required a new application to market—now called Lumizyme. The FDA’s decision on whether or not to approve Lumizyme for Pompe disease will be handed down by the end of the week.

 

Finally, NeurogesX (NGSX) expects a decision from the FDA on its drug Qutenza, a cutaneous patch for the treatment of peripheral neuropathic pain in non-diabetic adults, alone or in combination with other pain products. The company had been struggling to stay listed on NASDAQ until June when it licensed commercial rights for Quetenza for 27 countries in Europe, the Middle East and Africa in return for $42 million plus $7 million for a license option for a liquid formulation of the drug. The company’s shares were trading at $7.25 a share at the close of the week, below their midsummer high of $9.20.