Protalix BioTherapeutics (PLX) Gets Huge Opportunity Due To Genzyme's (GENZ) Plant Shut-Down

Recent news of a plant shutdown at Genzyme's (Nasdaq: GENZ) Allston Landing manufacturing facility could bode well for small-cap biopharmaceutical company Protalix BioTherapeutics, Inc. (NYSE: PLX).

Today, Protalix BioTherapeutics announced that it was approached by the FDA and asked to consider submitting a treatment protocol for the use of prGCD in patients with Gaucher disease in order to address an expected shortage of Genzyme's Cerezyme.

Shares of Protalix BioTherapeutics are reacting positively to the news, currently up 14%.

Gaucher disease is a rare and serious lysosomal storage disorder in humans with severe and debilitating symptoms. Protalix BioTherapeutics' prGCD is a proprietary plant-cell expressed recombinant form of glucocerebrosidase and is currently the subject of a Phase III clinical trial for the treatment of Gaucher disease.

The FDA indicated to the Company that it believes the Company’s development program for prGCD satisfies the regulatory criteria required to supply prGCD for expanded access to patients under a treatment protocol.

Under current FDA regulations, a treatment protocol may be submitted for a drug that has not yet been approved for marketing but is the subject of clinical development for a serious or life threatening disease for which no comparable or satisfactory alternative drug or therapy is available.

The Company and the FDA are discussing the parameters of a proposed treatment protocol which would allow an increased number of patients with Gaucher disease to have access to prGCD. The Company expects to submit a treatment protocol to the FDA for its review as a supplement to the Company’s current Investigational New Drug (IND) application for prGCD.

Analysts at Thomas Weisel said the treatment protocol could allow temporary market access of prGCD and, more importantly, could allow some physicians to gain comfort with switching patients on Cerezyme to prGCD, which is expected to enter the market at a 20-30% discount to Cerezyme.

That said, Thomas Weisel doesn't see more than a niche role for prGCD in the U.S. although non-U.S. it could play a bigger role.. The firm said, "We continue to expect that Protalix’s prGCD and Shire's Velaglucerase to have limited penetration in the U.S. market as Genzyme manages the relationships with prescribing physicians and Gaucher patients well. However, we believe that competitive pressure in ex-U.S. communities with a high prevalence of the disease, such as Israel and Brazil, and more influenced by pricing pressure could see a marginally higher rate of penetration."

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