Protalix BioTherapeutics Inc. (PLX:US) and Pfizer Inc. (PFE:US) failed to win European Union backing for a medicine to treat a rare genetic ailment because a competing treatment from Shire Plc (SHP) has exclusive market rights for 10 years. Protalix fell the most in more than six months.
Taliglucerase alfa shouldn’t be allowed on the market because it works in the same way as Shire’s Vpriv, the European Medicines Agency’s Committee for Medicinal Products for Human Use said in a statement today. Medicines for rare diseases, known as orphan drugs, are expensive to develop and have small patient populations, so drugmakers are given exclusivity as an incentive to develop them.
“From a commercial standpoint, this is obviously a setback that we will need to address,” Protalix Chief Executive Officer David Aviezer said on a conference call with analysts. “We will continue to do whatever we can to eventually try to reverse this refusal.” Pfizer’s legal team is “looking into different options,” he said.
The decision is a setback for Carmiel, Israel-based Protalix, which won approval for the product from the U.S. Food and Drug Administration on May 1 under the name Elelyso. The treatment, Protalix’s first drug on the market, helped patients with Gaucher disease in clinical trials, and side effects were similar to those of other enzyme replacement therapies, the London-based drug agency said today.
Protalix’s U.S. shares (PLX:US) fell 6.7 percent to $6.16 at 4 p.m. in New York. The shares have gained 25 percent this year, giving the company a market value of $566.1 million. Pfizer rose less than 1 percent to $22.73.
The drug is a plant-based medicine that replaces an enzyme missing in people with Gaucher disease, a genetic ailment that affects as many as one in 50,000 people in the U.S., according to the National Human Genome Research Institute. Sanofi (SAN) also sells a drug for Gaucher called Cerezyme.
Gaucher disease can cause fat to build up in the liver, spleen, bone marrow and nervous system. About 1 in 14 individuals of Ashkenazi Jewish ancestry carries the mutated gene that can cause the illness, and as many as 1 in 500 present a form of the disorder.
“While we are disappointed with the CHMP’s recommendation, we are encouraged that the committee gave a positive risk- benefit assessment,” Diem Nguyen, Pfizer’s general manager for biosimilars, said in a statement. “The recommendation was based solely on orphan market exclusivity and not the safety and efficacy profile of taliglucerase alfa. Pfizer will continue to work with relevant stakeholders to determine appropriate next steps.”
Pfizer unsuccessfully sought an exemption from the exclusivity rule based on several factors, the company said. There was no good evidence that Elelyso was clinically superior to Vpriv, or that Vpriv was in short supply, both of which are criteria for exemptions, the agency’s drug-review committee found.
Drugs for Gaucher and another illness, Fabry disease, have been in short supply in the past couple of years because of a 2009 virus contamination at a Genzyme Corp. plant in Allston, Massachusetts. Sanofi acquired Genzyme, the maker of Cerezyme, last year, and has said production levels are increasing. Protalix thought it could win an exemption for Elelyso because of the shortages, CEO Aviezer said.
The European Commission usually adopts the committee’s advice. Shire’s Vpriv was approved in Europe two years ago.
Pfizer, the world’s largest drugmaker, paid $60 million for rights to sell taliglucerase and agreed to pay Protalix an additional $55 million if the drug passed certain regulatory hurdles, according to a December 2009 statement. New York-based Pfizer is entitled to 60 percent of the drug’s revenue under the agreement.
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