Amicus Therapeutics Building Rare Disease Treatment Strategy; BofA Initiates Buy

Bank of America's Peter Stapor initiated a buy rating on

with a price target of $10.00. The leading drug in the company's portfolio is migalastat (Galafold) for a rare disease (Fabry Disease) which results in enzyme deficiency and leads to organ failure in several cases. Migalastat is a convenient alternative to other treatments as it can be taken orally. It is pending approval from Europe's Committee for Medicinal Products for Human Use. The acquisition of Scioderm added SD-101 (Zorblisa) to their portfolio. Zorblisa is in its phase three trial. It is being developed to treat Epidermolysis Bullosa (EB) a rare disease that affects infants and results in fragile skin and blisters and open wounds. These symptoms increase risk for complications such as sepsis and even squamous cell carcinoma. There are no approved treatments and Zorblisa would be able to corner this market quickly. Its "phase 2b data was encouraging" and "doctors indicate Zorblisa could have wide use" Stapor noted. FOLD is developing a compound to treat a third rare genetic disease (Pompe). Phase ½ are expected this year. Amicus Therapeutics is building a strong platform in rare disease treatment and with few to no drugs to compete against the upsides of this strategy seem large.