FDA-Approved ASO Therapies in 2017

As of 2017, there have been six FDA-approved antisense oligonucleotide (ASO)-based therapies on the market. These are all for non-brain diseases: diseases of the retina (2), liver (1), and three others. Number six (spinal muscular atrophy) is the closest we get to the brain. As I mentioned earlier, Ionis Pharmaceuticals is in the early stages of an ASO for Huntington’s disease.

Common points: they all are ameliorative treatments, not cures or preventatives; they are super-expensive, soon to break the USD one million mark, yearly.

Are ASOs evolving and becoming better? There’s no financial incentive in the U.S. to roll a drug more often than the 20 years after which its patent expires. Most FDA-approved ASOs haven’t been around long enough to have a history beyond 20 years; the only one that nearly has was discontinued. Who knows how the landscape will evolve?

1: fomivirsen

  • https://en.wikipedia.org/wiki/Fomivirsen
  • Brand name: Vitravene.
  • FDA approval: 1998-08-26.
  • Treatment for cytomegalovirus (CMV) retinitis.
    • An acquired (not genetic) disease.
    • Prevalence: maybe 100 per 100,000 (20 times as prevalent as SCA).
  • How administered: intraocular injection.
  • A treatment not a cure or preventative.
  • Discontinued because of low money-making potential, but its significance remains as being the first ASO-based drug on the market.

2: pegaptanib

  • https://en.wikipedia.org/wiki/Pegaptanib
  • Brand name: Macugen.
  • FDA approval: 2004-12-17.
  • Treatment for macular degeneration (wet AMD).
    • There are genetic risk factors.
    • Prevalence: 2,400 per 100,000 (500 times as prevalent as SCA).
  • How administered: intravitreal (eye) injection, every six weeks.
  • A treatment not a cure or preventative.
  • Cost: USD 46,000 per year.

3: mipomersen

4: defibrotide

  • https://en.wikipedia.org/wiki/Defibrotide
  • Brand name: Defitelio.
  • FDA approval: 2016-03-30.
  • Treatment for veno-occlusive disease, a liver disease.
    • An acquired (not genetic) disease.
    • Treats complications with chemotherapy or stem cell transplants.
    • Prevalence: unknown.
  • How administered: intravenous infusion, daily for 21-60 days.
  • Cost: USD 156,000 for 21 days.

5: eteplirsen

6: nusinersen

Leave a Reply

Your email address will not be published. Required fields are marked *