Minor Delay, Major Missed Opportunity

"Just realize that you're investing without being able to see the full landscape." Those were my parting words when I previewed the FDA decision for Protalix BioTherapeutics (AMEX: PLX  ) and Pfizer's (NYSE: PFE  ) Gaucher disease drug, Uplyso.

Looks like that landscape includes some plants that will need a little more water and tender loving care before the FDA approves Uplyso. The companies announced today that the agency wants more information before it'll OK the drug.

As I suggested might be a problem, the FDA is concerned about the manufacturing of Uplyso. The drug is made in plant cells, rather than the traditional mammalian cells with which the FDA has more experience. While in uncharted territory, the agency will be extra cautious to ensure that the manufacturing is up to snuff.

The agency also wants data on the crossover and long-term extension studies that are already ongoing. Assuming the data that has been collected to date is sufficient, it's just a matter of formatting that information and submitting to the agency.

Neither issue sounds like a major delay, but management wasn't willing to give a timeline before speaking with the FDA about exactly what it wants. I'd guess a few months to get the data together plus a six-month review process. Uplyso could be on the market by the end of the year.

The delay isn't the end of the world, but the companies really missed their opportunity to pounce. Genzyme (Nasdaq: GENZ  ) is getting Cerezyme production back to full capacity after its manufacturing train wreck, and now that the drama with sanofi-aventis (NYSE: SNY  ) is worked out, there should be fewer distractions. And Shire (Nasdaq: SHPGY  ) also has a new Gaucher drug, Vpriv, that will continue to establish itself while Protalix and Pfizer wait for an approval.

Better get pruning Protalix and Pfizer.

Interested in keeping track of Protalix as it tries to gain FDA approval? Click here to add it to My Watchlist, which will help you keep track of all our Foolish analysis on Protalix.

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Fool contributor Brian Orelli, Ph.D., doesn't own shares of any company mentioned in this article. The Fool has a disclosure policy.


Read/Post Comments (3) | Recommend This Article (1)

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  • Report this Comment On February 25, 2011, at 4:49 PM, foxdror wrote:

    The FDA could ask all tthose requests before,the last date for approval. This raise a questionon on the real purpose of the delay. It is the same FDA that approeved the use of PLX Uplyso.when Genzyme could not. It is clear that the FDA see that the Uplyso is better from the competition. The numbers are there tested. The Uplyso is already helping many around the world. Taking all the information into account shows that whoever decided to postpone the approeval has some data that can not be shared with others. It has happaned before and unfortunatly canhappan wherever people are involvrd.

  • Report this Comment On February 25, 2011, at 7:39 PM, steven107 wrote:

    This is the type of information that once FDA decides they are going to need it they can at that very moment make a request of the company for the information and the company can submit it via a minor/major amendment. There is no reason why the FDA needs to wait until the PDUFA Date to make this request. Perhaps they need to hire more staff and ask for a bigger budget, but this is rediculous. Glad I have stopped holding through PDUFA Dates. I do not trust FDA to do anything right. I do not subscribe to conspiracy theories, for no other reason than these types of problems can be explained by idiocy and incompetence why bring conspiracy into the mix (Occam's Razor). (assuming I spelled that right).

  • Report this Comment On February 25, 2011, at 11:33 PM, FarmaBiz wrote:

    PFE is more interested in the forest than just this one tree. In order to become a biologics power they need some edge. The cost savings of this development technique will allow follow on products (especially biosimilars and biobetters) combined with lower sales rep costs to maintain their profit margins in percentage terms and use volume to maintain and grow in absolute $ terms. Add in a weak dollar and explosive foreign growth (there will never be a reduction in demand for biopharna products) and you have the makings of a killer profit machine. Just what Dr. Read has prescribed; by the end of this year is de minimus.

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