Drug development: Ignore polymorphism at your peril

The consequences of Abbott Laboratories'granulation, melting, spray drying, compression and
antiretroviral drug Ritonavir, used to treat HIVmilling that are required to produce the final dosage
infection and AIDS and problems with polymorphismform. 
has yet to be universally understood.  AvoidingX-Ray Powder diffraction and Raman spectroscopy
action has not yet widely been taken onare the two workhorse methods used to evaluate
polymorphism by the global pharmaceutical industry,the presence and amount of different polymorphic
so it is worth repeating the story. forms found during screening.  Differential Scanning
In 1996 Abbott launched on the market an effectiveCalorimetry (DSC), Differential Thermal Analysis
protease inhibitor Norvir® that had cost the(DTA), Dynamic Vapour Sorption (DVS) and hot
company in excess of $200 million to develop.  Thestage microscopy are additional specialised techniques
drug was formulated as an encapsulated ethanolused to characterise different polymorphic forms.
water solution.  In the summer of 1998, supplies ofAll is not doom and gloom as the polymorphism
the drug were interrupted by the appearance of aproperties of a chemical substance are patentable. 
new crystal form (polymorphism) at a plant in theA new polymorphic form therefore may be used to
USA and then later at a plant in Italy.  This new,extend the patent life of a drug.  A counter to this
more thermodynamically stable polymorphic form hadis that a new polymorphic form with advantageous
very different physical properties than the earlierproperties may be able to supersede an existing drug
material and Abbott was forced to withdraw theand effectively ‘bust' the original patent.
drug from sale. The new form failed dissolution testsIt's not surprising to learn that manufacturers &
and precipitated out within the capsules. Thedevelopers of generic drugs actively pursue new
company lost an estimated $250 million in sales aspolymorphic forms of patented drug substances. 
well as hundreds of millions of dollars trying toThis can be a highly litigious area.
recover the original polymorph while the product wasNational regulatory authorities require that all
off the market.  No doubt many AIDS suffererscompanies register the precise polymorph of any
were not helped by the product's absence.  Whatnew drug.  Moreover, manufacturers need to
appeared to have happened was that a degradationdemonstrate that each polymorph is stable and can
product obtained during manufacturing had initiatedbe reliably reproduced
the appearance of a second crystalline form, aMany small pharmaceutical companies do not intend
second polymorph. taking their drug candidates all the way to
So what is polymorphism? It is simply a differentcommercialisation themselves but to seek to license
arrangement a molecule might adopt in a crystal. at an earlier stage.  This arises because of the
Most drug molecules are crystalline.  That is, theconsiderable increase in costs as a drug moves
molecules pack together in a particular regular way.through the clinic. If a polymorph screen is also
Some molecules, perhaps most, are able to packincluded then the package is certainly stronger and
together in more than one way and thus give rise tothe licensor can expect a further premium.
different polymorphs.  A pair of polymorphs mightDuring pre-clinical development the quantity of an API
have very different physical properties.  Over 50%that is available for studies is usually very low. 
of all Active Pharmaceutical Ingredients (APIs) exist inScreening a wide variety of solvents and conditions
at least 2 polymorphic forms.needs to be conducted for that reason on a very
During drug development, an initial scoutingsmall scale.  Systems that can handle multiple
polymorphism screen is designed to find a stableexperiments at the milligram scale are best.  At
non-solvated form with good properties.  A latersome labs automated systems can handle up to 96
comprehensive polymorphism screen is to find aswell plate formats and conduct experiments at
many forms as possible in order to exhaustively0.5-2mg scale.  Thus the total amount of API used
cover the Intellectual Property space.  Continuousfor an initial screen can be a modest 50-200mg.
monitoring is needed throughout development inTypically, a salt selection project will precede a
order to ensure continued control of polymorphism. polymorphism study:  once a salt is found that has
What can cause polymorphism changes? the most promising properties, it will be further
Crystallisation from different solvents may give risedeveloped, characterised and might be the subject of
to different crystal forms or solvates.  Extremes ofa further polymorphism screen during the normal
humidity or heat are among the more obviouscourse of pre-clinical development.
factors affecting polymorphism.  Changes inPolymorphism, in addition to complicating drug
polymorphism can also be induced as a consequencedevelopment also aids drug efficacy and can ensure
of several common stages of API processing such asthat any hard earned work is justifiably rewarded.