This morning in Shire's (SHPGY) earnings press release, Shire announced that it has received a complete response letter from the FDA regarding production of VPRIV drug substance produced at their Lexington facility. Shire's Vpriv is the second after Genzyme (SNY) to reach the market for Gaucher disease, and an important potential competitor to Protalix's (PLX) taligucerase.
- Shire has received a Complete Response letter from the US Food and Drug Administration ("FDA") regarding production of VPRIV drug substance at Lexington. Shire is working closely with the FDA to address their questions and resolve any outstanding issues to the satisfaction of the agency.
- Notwithstanding the ongoing discussions with the FDA, Shire continues to supply VPRIV to US patients through its existing approved US manufacturing facility at Alewife and has the capacity to meet the anticipated demand for VPRIV from existing and new patients both in the US and globally,recognizing that US inventory levels will be below target levels until the Lexington facility is approved by the FDA.
Even though Shire has received EMA approval of its Lexington manufacturing facility in February, which was necessary for Shire to increase its supply, the US inventory levels will be below target levels until the Lexington facility is approved by the FDA.
- On February 22, 2012 Shire announced that the European Medicines Agency's ("EMA") Committee for Medicinal Products for Human Use had approved the production of VPRIV in its new biologics manufacturing facility in Lexington, Massachusetts and this decision was adopted by the European Commission on March 26, 2012. Shire now has two EMA approved facilities - Alewife in Cambridge, Massachusetts, as well as the new Lexington facility - in which to manufacture VPRIV drug substance.
However, the unexpected CRL from the FDA announced by Shire this morning means that Shire will not yet be able to sell in the US anything produced in this facility, meaning their U.S. supply remains limited.
These news are positive for Protalix as the shortage in the US markets will continue. Sanofi's Cerezyme can't supply the demand in the US and the opportunity for Protalix to get approved is higher today; which means higher chances to gain larger market share in the US among those patients who are unable to receive full doses these days.
The shortage in the US and the worldwide issues around the Gauchger's drug supply may influence European regulators to make an exception to Vpriv's Orphan exclusivity and enable taliglucerase approval.
As I wrote in my latest article Protalix: What You Need To Know Ahead Of The May 1 PDUFA Date, I continue to believe there is a high likelihood of taliglucerase approvals in the U.S. and Europe, and today Shire's news gave Protalix's shareholder more reasons to hold through the FDA decision.
Protalix has high chances to get approval considering the following facts:
- It is an NDA resubmission and under SPA protocol.
- The FDA did not request long term or new clinical trials in the previous Complete Response Letter (CRL).
- Had good results of the Switch-Over trials presented at the 8th Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2012, held on February 8th in San Diego, California.
- The FDA is already allowing Protalix and Pfizer (PFE) to treat Gaucher's patients under a compassionate use program so the drug's efficacy and safety are not an issue.
- Have passed a "GMP" good manufacturing practices, audit of the Company's manufacturing facility was performed and it was found compliant with the FDA, the Israeli MOH, ANVISA and the IMB on behalf of the EMA.