Recently, the US Food and Drug Administration (“FDA”) accepted the New Drug Application (“NDA”) filed by Protalix BioTherapeutics Inc. (PLX) for its Gaucher drug, Uplyso (taliglucerase alfa). A response from the FDA should be out in the first quarter of 2011 (target date: February 25, 2011). Protalix has a marketing agreement with Pfizer Inc. (PFE) for the drug.
Uplyso is being developed for treating Gaucher disease under a Special Protocol Assessment (“SPA”) with the FDA. Protalix completed the submission of its rolling NDA on Uplyso to the US agency in April 2010. The submission came after the completion of a late-stage study for Uplyso, which enjoys orphan drug status in the US.
The rolling submission process allows companies to submit the NDA on drugs enjoying fast track designation in parts as and when data becomes available. This enables the review process to begin before the complete data package is available.
Competition Likely to Increase in Gaucher Market
Gaucher disease, a genetically transmitted disease, has an incidence of approximately 1 in 20,000 live births. Although Genzyme (GENZ) holds a leading position in the treatment of Gaucher disease, the company is still struggling to normalize its supply schedule for its Gaucher drug Cerezyme (“imiglucerase”).
Another important player in the market is Shire Plc’s (SHPGY) Vpriv (velaglucerase alfa). Vpriv is already available in the US where it received approval from the US FDA in late February 2010. Shire should be in a position to launch Vpriv in the EU by year-end. Meanwhile, the product launch in other countries should commence in 2011.
The approval of Uplyso expected early next year should intensify the competition in the Gaucher disease market, where the patient population is not very large. We note that both Vpriv and Uplyso were made available to patients under the FDA’s expanded access program due to the shortage of Cerezyme supplies.
Our Recommendation
Protalix is a Zacks #3 Rank (‘Hold’) stock, which indicates that it is expected to perform in line with the overall US equity market for the next 1 to 3 months. Our long-term Neutral stance on the company indicates that the stock is expected to replicate its short-term performance over the next 6 to 12 months. Consequently, we advise investors to retain the stock over the time period.
Our short-term as well as long-term recommendation on the stock is supported by the lack of estimates revisions by the analysts covering Protalix over the last thirty days. This indicates a lack of directional pressure on the stock.
Read the full analyst report on “PLX”
Read the full analyst report on “GENZ”
Read the full analyst report on “PFE”
Read the full analyst report on “SHPGY”
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