J&J, Pharmacyclics get CLL approval for Imbruvica

Johnson & Johnson ($JNJ) and Pharmacyclics' ($PCYC) new drug Imbruvica won the expanded approval it needed to go beyond a rare disease treatment and reach the blockbuster status it was conceived for. The FDA Wednesday gave Imbruvica accelerated approval for treating chronic lymphocytic leukemia (CLL) in patients who have received at least one previous therapy.

That follows the FDA's initial approval of the drug in November for treating patients with the rare blood cancer mantle cell lymphoma. That approval was also for patients who had already received at least one prior therapy. Mantle cell lymphoma affects about 2,900 people a year. How does that compare with CLL? The FDA said today that in 2013, 15,680 Americans were diagnosed with chronic lymphocytic leukemia and 4,580 died from from it.

That is a significant pool for a drug that is priced at more than $90 a pill--so is expected to run $98,400 per year for treatment. Because it takes more pills to treat MCL, a year's regimen is projected to run about $130,000. Of course, those figures are the price before discounts. Pharmacyclics will offer two months' worth of the drug for free to MCL patients who have trouble on the insurance-reimbursement side. Plus, the company is setting up copay assistance plans for patients who can't afford their share of the cost. Because of its improved benefits, it is expected to be in demand, putting pressure on payers to make it available under insurance plans. According to EvaluatePharma, analysts have forecast annual sales reaching $1.3 billion in 2018.

The signs for an expanded approval turned in the drug's favor last month when an independent panel stopped short a trial comparing Imbruvica with GlaxoSmithKline's ($GSK) Arzerra as a second-line treatment for CLL after Imbruvica showed significant improvement in both progression-free survival and overall survival. The FDA said today that an improvement in survival or disease-related symptoms has not yet been established, but the agency granted its accelerated approval on the condition that Pharmacyclics and J&J confirm the drug's benefits.

- here's the FDA announcement