Protalix BioTherapeutics, Inc. (PLX) was dealt a huge blow recently with the US Food and Drug Administration (FDA) issuing a complete response letter (CRL) for the company’s Gaucher disease candidate, taliglucerase alfa.
FDA Requests Additional Information
The FDA asked the company to provide additional information related to clinical and chemistry, manufacturing and controls (CMC). While the agency did not ask the company to conduct additional studies, it asked Protalix to provide data from the company’s switchover and long-term extension trials. Besides this, the FDA asked the company to submit information on testing specifications and assay validation. Depending on specific requirements, the Gaucher disease candidate’s approval could get delayed by a year.
The first-round approval of taliglucerase alfa would have been a major boost for Protalix, which has a strong marketing partner in Pfizer (PFE). The companies entered into a commercialization agreement in late 2009. Per the terms of the agreement, Pfizer has exclusive worldwide rights for the commercialization of taliglucerase alfa in all territories excluding Israel.
Gaucher Market
Genzyme’s (GENZ) Cerezyme holds a leading position in the treatment of Gaucher disease. Another important player in the market is Shire’s (SHPGY) Vpriv (velaglucerase alfa), which gained approval in the US as well as the EU in 2010. The potential approval of taliglucerase alfa will intensify the competition in the Gaucher disease market, where the patient population is not very large. We note that both Vpriv and taliglucerase alfa were made available to patients under the FDA’s expanded access program due to a shortage in Cerezyme supplies. Genzyme itself is currently working on an oral treatment for Gaucher disease.
Neutral on Protalix
We currently have a Zacks #4 rank (short-term “Sell” rating) on Protalix. The CRL for the Gaucher candidate is a major setback for the company. We expect investor focus to remain on the future regulatory path for the candidate. Protalix intends to conduct a meeting with the FDA shortly.
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