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 (2017/18).
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. ASOs were discovered in 1978, and the first ASO drug to be FDA-approved was 20 years later in 1998. 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
- https://en.wikipedia.org/wiki/Mipomersen
- Brand name: Kynamro.
- Developer: Ionis.
- FDA approval: 2013-01-29.
- Treatment for familial hypercholesterolemia.
- An autosomal dominant genetic disease.
- Prevalence: 3,000 per 100,000 (600 times as prevalent as SCA).
- How administered: subcutaneous injection, weekly (indefinitely?).
- A treatment not a cure or preventative.
- Cost: USD 176,000 per year.
4: defibrotide
- https://en.wikipedia.org/wiki/Defibrotide
- Brand name: Defitelio.
- Developer / manufacturer: Jazz.
- 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
- https://en.wikipedia.org/wiki/Eteplirsen
- Brand name: Exondys 51.
- Developer / manufacturer: Sarepta.
- FDA approval: 2016-09-16.
- Treatment for Duchenne muscular dystrophy.
- An X-linked recessive genetic disease.
- Prevalence: about 5 per 100,000 (about the same as SCA).
- How administered: intravenous infusion, weekly until no apparent benefit.
- A treatment not a cure or preventative.
- Cost: USD 300,000 per year.
6: nusinersen
- https://en.wikipedia.org/wiki/Nusinersen
- Brand name: Spinraza.
- Developer / manufacturer: Ionis / Biogen.
- FDA approval: 2016-12-23.
- Treatment for spinal muscular atrophy.
- An autosomal recessive genetic disease.
- Prevalence: about 10 per 100,000 (about twice as prevalent as SCA).
- How administered: intrathecal injection, four times in first month then every four months (indefinitely).
- A treatment not a cure or preventative.
- 2017-12-15. It’s better to begin treatment in babies.
- Cost: USD 750,000 for the first year, then USD 375,000 annually for one’s lifetime.
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