Here's an example today of an approval after Phase II under Breakthrough Therapy designation --
Pharmacyclics Drug Wins One Approval, Awaits Another
By AMY REEVES, INVESTOR'S BUSINESS DAILY
The Food and Drug Administration approved Pharmacyclics' (PCYC) cancer drug ibrutinib Wednesday, but it only cleared one of the two diseases the company had applied to treat.
The FDA approved the drug, now renamed Imbruvica, to treat mantle-cell lymphoma, a rare form of lymphoma currently treated with Celgene's (CELG) Revlimid and Takeda's Velcade.
It's only the second product to be approved under the agency's new "breakthrough therapy" designation, which allowed a decision to be made after only two phases of clinical trials instead of three. However, it didn't mention the part of Pharmacyclics' application asking for approval for chronic lymphocytic leukemia.
Analyst Michael Yee speculates the FDA might be waiting for data from an ongoing clinical trial called Resonate-1, expected in January, before it makes its final decision on CLL.
"Recall that we think inclusion of Resonate-1 data could actually be positive, as it would give physicians randomized data against (GlaxoSmithKline's (GSK) Ofatumamab to base prescribing decisions on, with better attribution of side effects to drug," he wrote in an e-mail to clients. He said the possible delay would be "immaterial" for 2014 estimates, and also noted that the FDA's comments had included no serious safety warnings.
The delay might also leave a bit more wiggle room for Gilead Sciences' (GILD) CLL candidate idelalisib, which reportedly did so well in its phase-three trial that Gilead ended it early last month.
The drug's launch and marketing will be handled by Johnson & Johnson (JNJ) division Janssen Pharmaceuticals.
As IBD reported last month, hopes that ibrutinib will be a blockbuster more than doubled the Pharmacyclics stock price this year, though it now trades 13% off its all-time high above 143 hit on Oct. 4. Pharmacyclics stock was down a fraction in morning trading in the stock market Wednesday, but the stock was up 4% in afternoon trading, after the FDA news broke.
