Langley’s Cam Penner’s efforts to help his 13-year-old son Doug, and others with Duchenne muscular dystrophy (DMD), hit a major road block this week.
According to Muscular Dystrophy Canada, DMD is an inherited disorder. The muscles become weaker as the patient gets older.
Doug is also the face behind Ride for Doug, an annual fundraising motorcycle ride organized by Cam.
On Nov. 24, Cam flew to Washington DC, where he addressed the Food and Drug Administration Advisory Committee about the impact that Drisapersen makes on Doug’s daily living.
“The study we have been on since 2011 has finally reached the stage where the FDA needs to either approve or reject it,” Cam explained in mid-December, as he awaited the ruling.
“If approved, other boys with DMD will be able to access the same medication Doug has been taking.”
On Thursday, Cam informed the Langley Advance that the FDA ruled not to approve the drug at this time.
“No reasons for this decision have been released,” Cam said.
The drug company BioMarin has issued a press release that current studies will continue for now.
This means Doug will still get his Drisapersen.
“Thank you BioMarin,” Cam said.
According to BioMarin, Duchenne affects approximately one in every 3,500 to 5,000 male children, making it the most common fatal genetic disorder diagnosed in childhood.
There is currently no FDA-approved therapy designed specifically to treat Duchenne.
For now, Cam said, he and others supporting the use of the drug do not know what is happening next.
“Drisapersen is under review by the EU equivalent of the FDA,” Cam said. “There is no timeline on if or when Canada will be included.”
While extremely disappointing for the Duchenne community, Cam said “our Medical Explorer will continue with his weekly treatments while we watch and pray that we continue to see its benefits.”
Prior to the ruling, Cam said a rejection “may spell the end to Drisapersen, and leave DMD boys with exactly zero approved treatments.”
As with all experimental medications, there have been risks, side effects, and confusing data, Cam explained.
“The FDA is wading through all of these issues and deliberating whether or not to allow access to this drug,” he said last month. “At this point in time, there are no other approved treatments for DMD. None.”
Link to the BioMarin press release here.