Protalix’s Uplyso for Gaucher’s disease not expected to see fast traction on FDA review

This article is provided to FT.com readers by BioPharm Insight—a news service focused on providing insight into the most price sensitive issues in the global pharmaceutical market. www.biopharminsight.com

--------------------------------------------------------------------------------

The FDA is unlikely to review Protalix Biotherapeutics’ (AMEX:PLX) Uplyso (taliglucerase alfa) for Gaucher’s disease sooner than the February 2012 PDUFA date, orphan drug experts and ex-FDA officials told Bopharm Insight.

They said there is not an urgent need for the drug considering that Sanofi (EPA:SAN) resolved shortage of its Gaucher’s disease drug, Cerezyme, earlier this year. In addition, the agency will likely place extra scrutiny on the chemistry and manufacturing issues on which it sought more details in a complete response letter (CRL) in February.

The company announced 17 August that the FDA had accepted its resubmission of its Uplyso new drug application. The FDA deemed the resubmission a Class II, or six-month, response and established 1 February 2012 as the PDUFA date.

FDA has asked for more data on the clinical trials and chemistry, manufacturing and controls (CMC) for the experimental treatment. Protalix said publicly the new submission addresses the FDA’s questions, including its request for clinical data from the company’s switchover trial and long-term extension trial.

FDA “is certainly going to be more cautious,” an ex-FDA compliance official and drug industry consultant said, considering the CRL issues and the fact that Uplyso is based on a novel technology, ProCellEx, a plant cell-based protein expression system. ProCellEx uses genetic engineering and plant (carrot and tobacco) cell culture technology instead of the traditional mammalian- or yeast-based systems. The agency likely assigned this PDUFA date to ensure it thoroughly reviews the clinical data and may even schedule a plant re-inspection, he said.

The consultant said he expects the FDA to wrap up a review soon after the New Year.

Due to the availability of Cerezyme, Uplyso is unlikely to get any special considerations, said Kathryn Mertes-Egeland, principal of drug consulting firm BRANDominance. She added that since Uplyso is a plant-based product and thus different from other competitors, “there may be more questions [by the FDA] around the CMC.”

An investment banker agreed there is a lack of urgency to approve the drug because Cerezyme already fills the niche. He also noted that the FDA has seemed to place additional scrutiny on manufacturing issues this year and thus the CMC issues at hand can lead to a longer review timeline.

A key issue, the banker said, is whether Protalix has tried to negotiate with FDA on the CMC concerns or simply heeded FDA’s rectification recommendations. The latter would allow for a quicker review, he added, but many companies try to offer the FDA alternative corrective actions and that means delayed review times.

Protalix president and CEO David Aviezer said in new reports, “We believe we have adequately addressed the requests that were outlined by the FDA in their complete response letter. We will continue to work closely with the FDA as it moves forward with the new drug application review.”

A second ex-FDA official and current drug industry member agreed the availability of Cerezyme dampens the FDA’s fervor in approving another Gaucher’s drug before the PDUFA date. He noted though that the CMC concerns may not be as significant as the other experts suggested, as the FDA has previously approved plant-based drugs.

--------------------------------------------------------------------------------

For more information or to inquire about a trial please email sales@biopharminsight.com or call Americas: +1 212-500-1384 or Europe: 44 (0)20 7059 6202