- Analyst Michael Yee of RBC Capital Markets has lifted his target price for Biomarin from $145 to $155. This positive rating is based on the conviction that drisapersen, whch Biomarin developed for treatment of Duchenne Muscular Dystrophy (DMD), has a good chance of winning approval.
- Drisapersen is an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to certain DMD patients.
- Drisapersen has been validated in the E.U. by the European Medicines Agency (EMA) and will soon be up for consideration for final approval, which could come as early as Q3 2016.
All currently available analyst reports on Biomarin have rated it a buy and none have rated it a sell. The reason: Excitement about the strong possibility of an approval for drisapersen in the U.S. and the E.U. In addition, Biomarin is also developing treatments for dwarfism and hemophilia, which also contribute to what Michael Yee considers a strong pipeline with a $4 billion revenue potential.
With respect to optimism about drisapersen, Yee pointed to strong safety data, a robust pool of patients (300-plus) and solid phase III data, in addition to a positive regulatory atmosphere in which the new FDA neuro division is working closely with Biomarin and taking into consideration input from advocates as well.