Pharming’s orphan drug receives second setback in Europe
pharmafile | March 26, 2008 | News story | Sales and Marketing |Â Â HAE, PharmingÂ
Dutch biotech company Pharming has suffered a blow, after its lead product Rhucin was rejected by European regulators for a second time.
The drug is designed to treat acute attacks of the genetic disorder Hereditary Angioedema (HAE), but has failed to gain authorisation due to a lack of evidence supporting its clinical benefits in repeat use.
Dr Francis Pinto, Pharming’s chief executive, said the company was disappointed with the decision, but that it will continue to address the unmet medical need of HAE patients.
He added: “The findings of the clinical studies have demonstrated clear evidence of Rhucin’s efficacy and safety in the treatment of acute HAE attacks. Rhucin acts quickly and none of the patients treated so far have experienced a relapse of an HAE attack or any serious treatment-related adverse events.”
Pharming specialise in the production of human proteins with healthcare potential, ranging from the treatment of genetic disorders to the repair of tissue damage during surgery and beyond. The proteins are produced through a combination of the company’s innovative technology and the milk of transgenic cows or rabbits.
Rhucin (recombinant human C1 inhibitor) is the most advanced drug in Pharming’s portfolio, which also has three products in pre-clinical trials.
HAE patients have on average of seven acute attacks per year and often have to be hospitalised during their attack for treatment of dehydration and pain management.
HAE has a prevalence of 1 in 30,000, and is characterised by acute attacks of painful and in some cases fatal swelling of several soft tissues – Rhucin is designed to neutralise the swelling.
But regulators found the available clinical data for the drug limited, with no evidence to say it was effective with repeat administration, which is the same conclusion that was drawn after Rhucin’s first submission in 2007.
The company has said it will again re-file its application, this time with additional data from a current US study, in the hope of gaining approval under fast-track review from the EMEA, which could happen by the end of 2008 if successful.
In the US Pharming intends to file Rhucin for marketing approval with the FDA later this year after the release of results from the same trial, which is expected in the three months time.
Pharming has also recently signed an agreement with EIP, a leading Turkish pharmaceutical company, for the sales, marketing and distribution of Rhucin in Turkey.
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