Sarepta and patient groups wanted to use the standard of historical controls, personal testimonies, and the presence of altered dystrophin in the body. On April 25, 2016, the Advisory Committee Panel voted against approval;. However, in June 2016, FDA requested for additional data from Sarepta to confirm findings of dystrophin production by eteplirsen. Janet Woodcock, director of the FDAs Center for Drug Evaluation and Research, overruled the panel, and FDA Commissioner Robert Califf deferred to her decision. Eterplirsen received accelerated approval on September 19, 2016.The European Medicines Agency reviewed the molecule in 2018, and refused to approve it.Following the approval of eteplirsen, two other drugs of a similar kind, golodirsen and viltolarsen received provisional approval from the FDA for the treatment of people with a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping as well as casimersen for exon 45 skipping. Society and culture
Economics
The US list price of eteplirsen is US$300,000 per year of treatment. The Institute for Clinical and Economic Review has found the drug not cost effective at the list price when the cost of one Quality-adjusted life year (QALY) was equal to US$150,000. References
External links
"Eteplirsen". Drug Information Portal. U.S. National Library of Medicine.
Monomethyl fumarate, sold under the brand name Bafiertam is a medication for the treatment of relapsing forms of multiple sclerosis, to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. It is taken by mouth. The most common adverse reactions are flushing, abdominal pain, diarrhea, and nausea.It was approved for medical use in the United States in April 2020. Pharmacology
Monomethyl fumarate alters the NFE2L2 (Nuclear factor erythroid 2-related factor 2) transcription factor. NFE2L2 (or NRF2) is a basic leucine zipper (bZIP) protein that regulates the expression of antioxidant proteins that protect against oxidative damage triggered by injury and inflammation. Several drugs that stimulate the NFE2L2 pathway are being studied for treatment of diseases that are caused by oxidative stress. Two precursors are also approved:

Dimethyl fumarate (brand name Tecfidera),
Diroximel fumarate (brand name Vumerity), approved by the FDA in October 2019. Society and culture
Names
Monomethyl fumarate is the international nonproprietary name (INN). References
External links
"Monomethyl fumarate". Drug Information Portal. U.S. National Library of Medicine.