Pfizer Gaucher drug rejected by CHMP
pharmafile | June 25, 2012 | News story | Sales and Marketing | Elelyso, FDA, Gaucher, Pfizer, Shire, Vpriv
A European committee has refused marketing authorisation for Pfizer’s Gaucher disease drug Elelyso, as a competitor drug has exclusivity to treat the disease.
The CHMP said it could not recommend the drug even though it has been proven to work, because of the ten-year market exclusivity that had been granted for Shire’s Vpriv, which was authorised in 2010 for the same condition.
Elelyso (taliglucerase alfa), which was recently approved by the FDA, was seeking a European licence for the long-term treatment of patients with type 1 Gaucher disease.
Market exclusivity for orphan medicines is given as an incentive for companies to develop medicines for rare diseases, which may otherwise not be developed due to the high costs and small patient populations.
But the exclusivity means that another medicine cannot be authorised for the same condition if it is similar to the medicine already authorised.
In this case, the CHMP concluded that Elelyso is similar to Vpriv, as they are both enzyme replacement therapies that work in the same way. The CHMP said Elelyso could have been used in Europe if it could show clinical superiority to Vpriv, or if there were supply problems with the drug.
But the Committee said there was ‘no good evidence’ that Elelyso would offer patients any important advantages over Vpriv, or that Vpriv is in short supply.
The Committee therefore recommended the refusal of the marketing authorisation.
“While we are disappointed by the CHMP’s recommendation, we are encouraged that the Committee gave a positive risk-benefit assessment,” said Diem Nguyen, general manager of biosimilars at Pfizer.
“Pfizer will continue to work with relevant stakeholders to determine appropriate next steps,” he added.
Gaucher disease is a rare inherited disorder, in which people do not have enough of an enzyme called glucocerebrosidase, which normally breaks down a fat called glucocerebroside.
Without the enzyme, glucocerebroside builds up in the body, typically in the liver, spleen, bone and some other tissues, causing a wide range of problems such as anaemia (low red blood cell counts), tiredness, easy bruising and a tendency to bleed, an enlarged spleen and liver, and bone pain and fractures.
Ben Adams
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