Doug Penner

Langley dad disappointed after bid for medication approval denied

The FDA chose to not approve a drug that, according to Cam Penner, makes a huge difference in his son Doug’s life.

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.

Just Posted

Heritage proposal for Aldergrove fire hall at a ‘standstill’

The Alder Grove Heritage Society faces a delay in knowing the fate of the fire hall due to tenants slated to live in the building until 2020.

Langley hosting high-flying fun at annual gymnastics tournament

Action runs Friday through Sunday at the Langley Events Centre fieldhouse.

BREAKING: Plecas won’t run in next election if legislature oversight reforms pass

B.C. Speaker and Abbotsford South MLA says he feels ‘great sympathy’ for Jody Wilson-Raybould

Langley Secondary students rehearse upcoming musical comedy

The Langley Secondary Musical Theatre Company presents Guys and Dolls on Feb. 22.

Guatemala mission offers hope, health

Peninsula team to visit remote villages, build a home, in Piedra Blanca

VIDEO: How much snow do you have?

Langley residents are being asked to share images of their snow to enter for a $20 coffee card.

Eight cases of measles confirmed in Vancouver outbreak

Coastal Health official say the cases stem from the French-language Ecole Jules Verne Secondary

Ontario police field complaints over Amber Alert for missing girl, 11, found dead

Some said the Amber Alert issued late Thursday for Riya Rajkumar disrupted their sleep

Former B.C. premier Gordon Campbell accused of sexual touching

Accuser went to police, interviewed by Britian’s Daily Telegraph

B.C. couple attacked with acid, slashed with knife in Vietnam

Warning, graphic images: Man has burns on 80 per cent of his body, slashed with knife

Northern B.C. First Nation clan says ancient tools found at pipeline work site

Archeologists from the Smithsonian Institute estimate one of the stones found dates back up to 3500 years

Man taken to hospital for smoke inhalation, burns after fire at RV park

Firefighters find man suffering from smoke inhalation, burns to face and hands: battalion chief

Wilson-Raybould resignation stokes anger, frustration within veterans community

Liberals have had three veterans-affairs ministers — Kent Hehr, Seamus O’Regan and Wilson-Raybould

Most Read