Nusinersen is an oligonucleotide drug approved by the FDA in 2016 for the treatment of spinal muscular atrophy (SMA). SMA is a rare genetic disease that affects the muscles used for movement, breathing, and swallowing. Nusinersen is the first and only approved treatment for SMA.
Nusinersen is administered through a spinal injection, and works by increasing the production of a protein called survival motor neuron (SMN) which is critical for the survival of motor neurons. By increasing SMN production, nusinersen can slow down or even stop the progression of SMA.
Nusinersen is an example of a new class of drugs called antisense oligonucleotides (ASOs). ASOs are a type of synthetic DNA or RNA molecule that can bind to a specific target RNA molecule and modify its expression. This makes ASOs a promising approach for treating a wide range of diseases caused by genetic mutations.
In addition to Nusinersen, there are several other approved oligonucleotide drugs on the market. These include:
- Eteplirsen (Exondys 51), approved for the treatment of Duchenne muscular dystrophy
- Inotersen (Tegsedi), approved for the treatment of hereditary transthyretin amyloidosis
- Patisiran (Onpattro), approved for the treatment of hereditary transthyretin-mediated amyloidosis
These drugs have shown promising results in clinical trials and offer hope for patients with rare genetic diseases. However, they are also very expensive and may not be accessible to all patients.
Nusinersen and other oligonucleotide drugs represent a new frontier in medicine, offering targeted therapies for rare genetic diseases. While these drugs are expensive, they offer hope for patients who previously had no treatment options. It is important for researchers and policymakers to continue to support the development of these innovative therapies and work to make them accessible to all who need them. A generic of Nusinersen would go a long way to making this therapy more accessible.