FDA shoots down Vertex's latest bid for Kalydeco expansion

Vertex Chief Medical Officer Jeffrey Chodakewitz

Vertex ($VRTX) has so far had a lot of success widening the patient pool for orphan drug Kalydeco. But in its latest quest for a label expansion, the FDA has stopped it in its tracks.

Friday, the Massachusetts biotech announced that U.S. regulators had handed it a Complete Response Letter over its bid to use the cystic fibrosis med in patients age 2 and older with one of 23 specific mutations in the CFTR gene. Now, the company will meet with the agency to hammer out a path forward.

Vertex based its application on preclinical data and on older results from a Phase IIa study. Within that trial, 19 of the 24 patients enrolled represented exhibited 8 of the 23 mutations the company sought approval to treat.

But as Vertex Chief Medical Officer Jeffrey Chodakewitz explained in a statement, "our intention with this submission was to rapidly bring Kalydeco to additional people with CF who we believe may benefit. We chose to pursue this approach given our strong belief in the science of CF and the well-established safety of Kalydeco across many different groups of people."

Kalydeco, the first and only treatment to treat the underlying cause of CF--a life-threatening genetic disorder that damages the lungs--has until now proved a hit with regulators. Since hitting the scene in early 2012 as a treatment for CF patients with one particular genetic mutation, the therapy has scored a series of new approvals that have dramatically expanded its patient pool--and its maker's revenue potential. Most recently, the FDA granted a go-ahead for new drug Orkambi--which combines Kalydeco with compound lumacaftor--to treat 8,500 eligible U.S. patients with the F508del mutation.

Courtesy of Vertex

Payers haven't been quite so enthusiastic, though, considering Kalydeco and Orkambi's high prices. After Vertex set a $259,000 price tag for Orkambi, a group of prominent CF specialists took its pricing fight public, calling the company's decision "egregious" and "not sustainable."

Vertex, though, has worked to justify its move by arguing that helping the world's CF population will take "very significant continued investment," a company spokesman said last summer. And beyond that, it's scaled up its patient services team; Last February, before Orkambi's approval, Chief Commercial Officer Stuart Arbuckle said it would help do so in order to "help providers and patients navigate the reimbursement process and also help with patient education and compliance."

- read the release

Special Reports: Top 15 Drug Launch Superstars - Kalydeco - Vertex Pharmaceuticals | Top 20 orphan drugs by 2018 - Kalydeco