Alnylam Receives Orphan Drug Status for Amyloidosis Drug | GenomeWeb

Alnylam Pharmaceuticals said this week that the US Food and Drug Administration has given orphan drug status to the company's investigational transthyretin-mediated amyloidosis ALN-TTR02 as a treatment for familial amyloidotic polyneuropathy, a common manifestation of the disease.

The designation provides a drugmaker with, among other things, extended market exclusivity for its product, tax credits, and marketing incentives.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

A trial upgrade to GenomeWeb Premium gives you full site access, interest-based email alerts, access to archives, and more. Never miss another important industry story.

Try GenomeWeb Premium now.

Already a GenomeWeb Premium member? Login Now.
Or, See if your institution qualifies for premium access.

*Before your trial expires, we’ll put together a custom quote with your long-term premium options.

Not ready for premium?

Browse our free articles
You can still register for access to our free content.

In PLOS this week: cell-free DNA from tumors is shorter than DNA from healthy cells, type 2 diabetes-related loci, and more.

Genetic analysis confirms that blood found on leaves collected near Marche-les-Dames some 80 years ago belongs to King Albert I of Belgium.

Researchers in the US begin to seek participants for the 1 million-person precision medicine cohort, the New York Times reports.

A new report indicates that women are underrepresented among US patent holders.