As we have discussed earlier, Shire’s (SHPG) LSD (or lysosomal storage disorders) portfolio consists of Elaprase, Replagal, and Vpriv. Elaprase is an enzyme replacement therapy indicated for Hunter syndrome (Mucopolysaccharidosis II). In developing markets, the drug is growing quickly due to the number of new patients while in mature markets it’s growing slowly. Shire expects Elaprase to grow in the mid-single digits in 2016. Rare disease companies such as BioMarin (BMRN) operate in this space.
Replagal is indicated for Fabry disease. The drug had normalized growth by 6% in 1Q16, and similar growth is expected from the drug in fiscal 2016. Replagal’s overall growth in 1Q16 was 12%, which was driven by large orders in Japan. Sanofi’s Fabrazyme competes with Replagal.
Vpriv is indicated for Gaucher disease. The drug saw flat growth following the launch of a new competitive oral drug therapy called Cerdalga from Sanofi (SNY). Vpriv sales were eroded by Cerdalga. Cerdalga is the most expensive drug in the space. It costs $310,250 per patient per year. Sanofi holds Cerezyme and Cerdalga under its Gaucher franchise. To understand Sanofi’s leadership in the space, you can refer to Rare Disease Portfolio Helped Sanofi to Offset Falling Diabetes Sales.
Pfizer’s (PFE) Elelyso shares the market with Cerezyme, Cerdalga, and Vpriv. Elelyso was launched at a significant discount to Cerezyme, which annually costs $200,000–$300,000 per patient in the US. The ProShares Ultra Nasdaq Biotechnology (BIB) offers diversified exposure to high risk equities. The fund invests 0.9% of its holdings in Shire.