Since 2016 there have been four FDA approved ASOs. Defibrotide...

Since 2016 there have been four FDA approved ASOs.

• Defibrotide (Defitilio) is for veno-occlusive disease, a rare liver disease. The cost is US$ 156,000 for 21 days. Defitilio earned Jazz Pharmaceuticals ~ US$40m in revenue last quarter.
• Eterplirsen (Exondys 51) is for Duchenne (DMD). It costs US$300,000 pa. Exondys 51 brought in US$78m for Sarepta last quarter.
• Nusinersen (Spinraza) is the standout performer. It’s for SMA and costs US$750,000 for the first year of treatment and $375,000 pa thereafter. It is already tracking $1bn+ pa sales for Biogen.
• Inotersen (Tegsedi) is owned by Akcea Therapeutics /Ionis and was only approved in October. It’s indicated for polyneuropathy ATTR, a condition in which Alnylam already has an approved drug. The list price for Tegsedi is US$450,000 pa.

I note that all of these drugs, despite their high prices, are considered to be “a treatment, not a cure or preventative”.

I also note the following in the 31 October update

‘Can our CPPs deliver a therapeutic cargo to a tissue/cell affected by a disease process in a manner that enables the correction of that disease’?

We will answer this question for multiple cargoes and in multiple tissues in therapeutic read-outs (both in vivo and ex vivo) in 2019.