The Pharmaceutical KnowledgeBase (PharmaKB.com) provides research and financial intelligence about companies and their approved drugs as well as drug candidates in clinical trials. In this monthly PharmaKB report, we provide an update about four novel drugs approved by the US FDA. By referencing new drugs as “novel” the FDA is confirming that the medicine being approved contains ingredients and/or a mechanism of action that the FDA hasn’t previously approved.
April 2023 – Novel FDA Drug Approvals
Tofersen
- Drug: Tofersen
- Trade name: Qalsody
- Therapeutic Areas: amyotrophic lateral sclerosis (ALS) MeSH D000690
- Manufacturer: Biogen
- FDA Approval Date: April 25, 2023 (Abbreviated New Drug Application)
- Classification: Oligonucleotide
- Drug Class: Antisense oligonucleotides
- Clinical Trials: https://www.pharmakb.com/drug-report/tofersen#clinical-trials
- Adverse Events: https://www.pharmakb.com/drug-report/tofersen#adverse
- Description: Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with ALS. It is administered as an intrathecal injection into the spinal cord.
- FDA News Release: https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene
