The pharmaceutical industry achieved 39 approvals in 2012 – the highest number for several years – triggering a much-needed wave of optimism.  But sadly approval is not a guarantee of either patient benefit or commercial success.  So DrugBaron looked a little deeper at the drugs behind the headline numbers.

For a start, only 35 of the 39 were drugs (two imaging agents were approved, together with a laxative for colonic imaging and a surfactant for pre-term infant lungs), and of the 35 only 15 targeted new mechanisms of action, with the rest offering, for the most part, minor benefits over already-approved therapeutic options.

Seven of the approved drugs offer treatment for conditions where there was no real previous options – and therefore represent unambiguous and significant patient benefit.  Most strikingly, though, five of these seven were in indications that are not just orphan but ultra-tiny.  Indeed, in the case of Raxibacumab, the antibody against anthrax toxin from Human Genome Sciences, the patient population might be zero in the absence of an outbreak of biological warfare.  Of the seven drugs for previously untreatable indications, only Arena’a anti-obesity drug Belviq and Fulyzaq from Salix, a drug to treat GI complications of HIV, have the potential to treat more than a few thousand patients.

The 2012 approvals were a massive wake up call for the industry – the question is, was anyone listening?

The eight drugs bringing new mechanism of action to a disease where treatment options already exist also have the potential to deliver real advances.  Some will: Pfizer’s jak inhibitor Xeljans will find a place in the treatment paradigm for rheumatoid arthritis, and the unheralded beta3 adrenoceptor antagonist Myrbetriq from Astellas will bring relief to the many with overactive bladder that is poorly controlled by existing alpha blockers.  But others, most notably the Factor Xa inhibitor Eliquis from BMS offers little or no advantage over the generic standard of care.

Of the remainder, some fifteen (more than a third of the total approvals) are oncology drugs, mostly more kinase inhibitors, many on accelerated approvals where patient benefit remains to determined.  Even those with a full dataset to support approval deliver only a month or two benefit on overall survival, and it has to be questioned whether they really offer sufficient value to justify their use at all (although among the oncology approvals the prospects for Xtandi in prostate cancer may be rosier).

Looking at sales projections, how many blockbusters are there here?   DrugBaron projects peak sales over $1billion a year for Stribild, which is likely to become first …

RxCelerate Ltd is an outsourced drug development platform based near Cambridge, UK. We specialize in delivering an entire road map of drug development services from discovery and medicinal chemistry through to formal preclinical development and clinical up to Phase IIa. In the last five years, we have witnessed dramatic changes in the drug development …