Let’s start from the basics. Doctors will always opt for convenience when prescribing therapies to patients, especially when you have multiple options available, even for rare diseases. In this case, the 3 existing therapies for Gaucher disease are injectable drugs from Protalix/Pfizer – Elelyso; Sanofi/Genzyme – Cerezyme; and Shire’s Vpriv. Convenience in this case means price, but would it change if one of these became an oral drug?
Most likely yes! But why is this important and why is the industry talking about this?
Because Protalix Biotherapeutics has recently announced that PRX-112 – its oral formulation for the injectable Elelyso – has shown promising Phase 1 clinical data and if successful this drug will threaten the market share of Shire and Sanofi in the space, whose 2012 sales figures were $307million and $859million, respectively. This new oral drug can even set a challenge for Protalix’s own Elelyso, which sales data has not been reported but a deal with Brazil’s Ministry of Health for the commercialization of the product is expected to reach $280million in the next few years. Where are the carrots in this story?
The secret of PRX-112 is genetically engineered carrot cells, which shields the enzyme and protect it from the digestive tract, so it can successfully pass it and attach to the mucosal lining of the intestine. If proven successful, this new delivery platform can serve for other ERTs for other diseases.
Added to that, convenience would also come from a 25% lower price for the new drug if compared with market leader Cerezyme, which is only possible due to the lower production costs stemming from major manufacturing advantages.
At the World Orphan Drug Congress USA 2014 next April in D.C. you’ll get the chance to hear other approaches by pharma and biotech who are shunning the status quo of the orphan drug industry.