Baillie Gifford-favourite Alnylam completes key US filing
US biotech star and Baillie Gifford stock pick Alnylam Pharmaceuticals has completed its US regulatory application for patisiran, a treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), putting the so-called RNA interference or “gene-silencing” therapeutic on track for approval in mid 2018. Patisiran may become Alnylam’s first marketed product and holds breakthrough therapy status with the FDA, which provides for an accelerated approval timeline. Pending regulatory approvals, Alnylam will commercialise patisiran in the US, Canada and Western Europe, with its partner Sanofi selling the product in the rest of the world, including Central and Eastern European countries. Separately, Alnylam has been granted a request to expand its orphan drug designation to cover ATTR amyloidosis, expanding its prior designation that was limited to familial amyloidotic polyneuropathy. Orphan drug designation is a scheme that provides various incentives to develop drugs for conditions that affect small numbers of patients and effectively allows companies to charge high prices in these rare conditions.
Shares in Alnylam have trebled this year to $128 on the back of positive results in the Apollo Phase III study, first disclosed in September and later presented at a conference in November. This showed patisiran ameliorated neurological impairment, improved quality of life and reduced disease symptoms and disability in hATTR amyloidosis patients with polyneuropathy.
The rise in Alnylam stock helped boost the share price of Baillie Gifford-managed Edinburgh Worldwide Investment Trust EWI by some 40% this year. Alnylam is the trust’s largest single holding (5.8% of NAV). Edinburgh Worldwide also has one of the highest exposures to healthcare at c26% outside the specialist healthcare/biotech funds and therefore may offer a way to obtain more diversified exposure to the otherwise volatile sector.
ALNY : Baillie Gifford-favourite Alnylam completes key US filing