PTC scores new Emflaza approval in DMD patients 2 and older

Duchenne muscular dystrophy (DMD) drug Emflaza had a controversial past under its previous owner, but its current captain, PTC Therapeutics, is forging ahead—and a new FDA approval could help.  

The FDA on Friday cleared Emflaza for DMD patients as young as two years old. Previously, the drug was approved to treat patients five and older. The new nod will allow PTC to promote Emflaza for use earlier in the disease—and it makes the PTC med the only one FDA-approved for DMD patients two and older.

Earlier treatment could deliver better results for patients, PTC Therapeutics CEO Stuart Peltz said in a statement. “We believe that treating patients as young as possible, when they still have a substantial amount of muscle, will have the greatest benefit for patients that are two years and older," he said.

Emflaza’s road hasn’t been smooth. The med, a decades-old steroid, won FDA approval under owner Marathon Pharma, which then set a $85,000 per year price. The same drug is available for $1,000 per year in some countries.

After an onslaught of criticism, including a congressional investigation, Marathon sold the med to PTC for $140 million and shuttered its own operations. 

RELATED: PTC in the hot seat again over price hikes on DMD med Emflaza 

PTC priced the med at $35,000 after discounts, but some market watchers still questioned the new sticker. Cantor Fitzgerald analysts have predicted the drug’s sales will peak at $167 million in 2023.

The company has been "diligently preparing" for the new launch, Chief Operating Officer Marcio Souza said on a conference call last month. In the first quarter, PTC reported about $18 million in Emflaza sales, falling far short of analyst estimates of $35 million. The company recently switched specialty pharmacies, which hurt sales during the period, executives said during the call.

Overall, the company expects $285 million to $305 million in DMD sales this year. Aside from Emflaza, PTC Therapeutics markets DMD med Translarna in Europe; altogether, its DMD franchise brought in $53.6 million in the first quarter.

RELATED: Marathon, under heavy fire for Emflaza pricing, makes surprise deal to sell drug for $140M-plus 

PTC also sells Tegsedi, which treats polyneuropathy of hereditary transthyretin-mediated amyloidosis, in Latin America. It has several meds in its pipeline and is pushing for $1.5 billion in global sales by 2023.