The U.S. Food and Drug Administration approved a drug Monday that will give some boys suffering from a rare disease access to the potential life-saving drug, but some claim the approval could have come earlier possibly saving some who have already died.
The FDA announced it fast tracked approval of eteplirsen to treat young boys with the rare Duchenne muscular dystrophy.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research, in a prepared statement.
“In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval,” said Woodcock.
While one group fighting for better access to developmental drugs was happy eteplirsen was approved, they claim the drug should have been approved earlier.
“The Abigail Alliance for Better Access to Developmental Drugs is happy that eteplirsen is finally approved for young boys with Duchenne muscular dystrophy. However, this drug should have been approved three plus years ago, when Duchene patient advocates and the Abigail Alliance started pushing for its approval,” said Frank Burroughs, the president of the alliance, in a open letter today to the FDA and Woodcock.
Burroughs co-founder of the Abigail Alliance, named after his daughter, advocates for wider access to developmental drugs. His daughter died at 21 after exhausting all FDA approved drugs and her access to Erbitux was denied. That drug, which showed promising signs in early clinical trials, was approved three and a half year’s after Abigail’s death.
“Every drug for cancer and other serious life-threatening illnesses that the Abigail Alliance pushed for much earlier approval of in our 15- year history is now approved by the FDA,” said Burroughs. “Not one drug we pushed for failed to make it through the clinical trial process. The current total is 29 drugs! Five of the top ten most widely used cancer drugs are on the list!”
Duchenne muscular dystrophy is a debilitating muscle disease in boys, who are wheelchair bound by age 12 and do not historically survive beyond their mid-twenties. In late stages of the disease, heart and breathing muscles fail, according to the Cure Duchenne Organization.