On September 19, 2016, Exondys 51 was granted Accelerated Approval by the Food and Drug Administration (FDA), based on dystrophin as a surrogate endpoint, for the treatment of Duchenne. Exondys 51 is the only disease modifying treatment for Duchenne approved in the United States that can replace dystrophin, the protein patients with Duchenne are missing.
Exondys 51 is not approved by the European Medicines Agency (EMA). Exondys 51 treats approximately 13% of the Duchenne population who are amenable to the skipping of exon 51 on the dystrophin gene.
In February of 2017, the FDA approved Emflaza, a corticosteroid similar to Prednisone, for the treatment of Duchenne. Deflazacort has been imported by the Duchenne and Becker communities for decades. Deflazacort still can be imported from pharmacies outside the US with a physician's prescription. Emflaza is the only steroid approved for the indication of Duchenne.
If you or someone you know is interested in beginning treatment of Emflaza, please speak with your physician about obtaining a prescription, or contact PTC Cares.