Top 5 Most Surprising Pharma Regulatory Agency Rejections

Following the CHMP's recent negative opinion of Pfizer's Xeljanz, I thought I'd take a look at some of the regulatory agency decisions that have taken some people by surprise over the years.

XeljanzRejected by: Committee for Medicinal Products for Human Use (CHMP)

rheumatoid arthritis xeljanz rejection

Pfizer's rheumatoid arthritis drug Xeljanz was handed a negative opinion by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) in late April 2013. The CHMP held the view that Xeljanz didn't demonstrate a favourable risk:benefit profile. The decision was particularly surprising, as the data package it has submitted to the EMA was the same as it had submitted to the FDA – who approved the drug last November.



ContraveRejected by: Food and Drug Administration (FDA)

contrave orexigen (tobyotter)Perhaps it shouldn't have been that much of a surprise – given the FDA's track record of anti-obesity drug rejections – but in February 2011, Orexigen Therapeutics were sent reeling after the FDA rejected their prescription diet pill Contrave. Hopes had been held high that Contrave would be the first new diet drug in a decade, but the FDA refused approval until additional safety studies had been carried out to investigate Contrave's side effects on the heart. The rejection came after an FDA advisory panel had previously backed approval for the drug by 13 votes to 7.


Fycompa Rejected by: German Federal Joint Committee (G-BA)

epilepsy fycompa rejection (cloois)Eisai were reportedly "appalled by the G-BA's ruling" in March 2013 that the additional benefits of their antiepileptic drug Fycompa were unproven. Fycompa was a first-in-class drug for the treatment of uncontrolled partial epilepsy, but the Committee ruled that the additional benefits were unproven when compared to GSK's Lamicta and J&J's Topamax. Eisai said it believed the G-BA “failed to adequately interpret the proven patient-relevant benefits substantiated in the submitted benefit dossier and to responsibly recognise the innovative nature of the new drug in a clinical setting with a highly unmet medical need”, and the rejection came despite Eisai having "”diligently developed the benefit dossier following scientific advice from the G-BA”.


BrilintaRejected by: Food and Drug Administration (FDA)

brilinta blood astrazeneca fda (m.mate)In December 2010, AstraZeneca's blockbuster blood thinning drug was rejected by the FDA, asking for more analysis of a key clinical study. The news was a bit of a shocker, as the drug was already approved in Europe, and an FDA advisory panel had previously voted 7 to 1 in favour of recommending marketing approval. The rejection came as AstraZeneca's patents were due to expire on big sellers Nexium and Seroquel , and Plavix generics were about to hit the market.

Tresiba Rejected by: Food and Drug Administration (FDA)

tresiba novo nordisk fda (Jill A. Brown)It had already secured regulatory approvals in Europe and Japan, as well as a positive 8-4 vote from the FDA advisory committe, but Novo Nordisk's diabetes drugs Tresiba (insulin degludec) and Ryzodeg (insulin degludec/insulin aspart) did not receive approval from the FDA. The regulator had decided that it required additional proof of the products' safety via a cardiovascular outcomes trial.



What do you think? Were you not surprised by some of these decisions? Are there any surprising decisions you think should be on the list? You can join our discussion on LinkedIn, or leave a comment below. Want more from Total BioPharma? Sign up to our newsletter – it doesn't cost anything and only takes a minute.

Of course, all of these drugs made it through the late stages of clinical development – but if you want to know more about early stage development and avoiding the expense of carrying ineffective novel agents into full scale clinical development, you might be interested in attending Exploratory Clinical Development World Europe, 4-6 June 2013, London.

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