Belgian drugmaker UCB suffered a major blow last week after European advisors upheld a negative opinion on Cimzia for Crohn’s disease, just days after the firm announced it was temporarily recalling its Parkinson’s disease patch Neupro.

The decision follows an appeal by UCB after the European Medicines Agency’s Committee for Medicinal Products for Human Use originally advised turning down its application to market Cimzia (certolizumab pegol) - the first and only PEGylated anti-tumour necrosis factor alpha drug - for Crohn’s back in November last year.

According to the CHMP, although certain issues had been resolved during the appeal process, including fears over a possible increased risk of bleeding associated with the product’s use, it remains concerned over the drug’s general safety and effectiveness profile, and so concluded that its benefits to patients do not outweigh the potential risks in this setting.

But UCB will likely be feeling particularly disappointed with the decision, given that it recently released six-week data from a 539-patient Phase IIIb trial showing that the drug is effective in treating patients with the disease who are intolerant or no longer responding to infliximab. At week six of the 26-week trial, 61% of patients receiving subcutaneous injections of Cimzia 400mg at weeks zero, two and four had achieved the primary endpoint of a decrease in Crohn's disease, with 39% in remission.

The second rejection by the Committee certainly represents a major setback to the firm as Cimzia, which is approved for use in Switzerland, has been touted as a blockbuster that could help plug the gap in sales resulting from the loss of patent protection on its biggest earners – the allergy drug Zyrtec (cetirizine) and the antiepileptic Keppra (levetiracetam) – in 2007 and 2009, respectively.

Pharming stock drops on thumbs down
Meanwhile, shares in Dutch group Pharming plunged before the weekend after it said it expected the CHMP to uphold its negative opinion for its flagship hereditary angioedema drug Rhucin.

The group’s shares ended up closing down nearly 25% on Thursday after a day of frenzied trading in Amsterdam following the news, although it maintained that the Committee “does not have a specific concern on the safety and efficacy data” and reaffirmed its commitment to getting the drug on the market.

According to Pharming, the CHMP felt there was insufficient evidence in the file to confirm the clinical benefits of Rhucin (recombinant human C1 inhibitor) in repeat use and, in particular, to address the potential for undesirable immune responses following repeated administration.

But the company has refused to take the decision lying down, promising to re-submit the drug when new data from a recently completed North American study become available in the second quarter. Furthermore, the company said it plans to request a speedy review of the re-submission, and that it will continue to pursue registration in other markets in the meantime.

“We are disappointed by the opinion of the CHMP but will continue to work with the EMEA to address the unmet medical need of HAE patients,” commented Francis Pinto, Chief Executive Officer of Pharming, and he said the firm remains committed “to making Rhucin available to European HAE patients by re-filing our Marketing Authorisation Application with additional data and obtaining approval under an expedited review from EMEA.”