ViewPoints: Will the FDA send out message on breakthrough therapy status with speedy approval for Johnson & Johnson, Pharmacyclics' multi-billion dollar candidate ibrutinib?

In a move that will further fuel suggestions that the FDA has become much more receptive to drugs demonstrating real innovation, Johnson & Johnson and Pharmacyclics surprised many by announcing on Wednesday that they have filed their experimental cancer treatment ibrutinib with the administration.

The two companies had indicated that the application – for previously treated patients with chronic lymphocytic leukaemia (CLL)/small lymphocytic lymphoma (SLL), and previously treated patients with mantle cell lymphoma (MCL) – would be filed before the end of Q3, although few expected this to occur in early July. In a note to investors, Goldman Sachs analyst Navdeep Singh suggested that filing in CLL (based on Phase II data) comes a year ahead of schedule and according to management was encouraged by the FDA.

Insight, Analysis & Opinion

Ibrutinib is no stranger to the wave of regulatory momentum that the FDA has been keen to instigate in recent months. The drug has received three separate breakthrough designations from the administration; for CLL or SLL, relapsed or refractory MCL and Waldenstrom's macroglobulinaemia.

With the merits of breakthrough designation still being hotly debated by industry commentators, the speed at which ibrutinib now progresses through the regulatory arena is likely to be viewed as an important benchmark for other products that have been awarded breakthrough status.

Ibrutinib will not be the first product with breakthrough designation to receive approval (Vertex Pharmaceuticals' Kalydeco has already achieved this), although it is likely to be the first product with this status assessed by the FDA since the administration has become more vocal about the benefits that breakthrough designation is designed to provide.

The FDA – in recently talking up the merits of breakthrough status – may also have put itself under some pressure. Analysts are now speculating that an earlier than expected application for ibrutinib could see the product approved by the end of 2013 (i.e. within five months).

There is mounting evidence to support this bullish view. The Street's David Sobek – who predicted a 2013 approval for ibrutinib back in May – cites the FDA's trend for "reviewing cancer drugs with alacrity" as a reason to be confident, pointing to the approvals of Ariad Pharmaceuticals' Iclusig (chronic myeloid leukaemia; December 2012) and Onyx Pharmaceuticals' Kyprolis (multiple myeloma; July 2012) as recent examples.

However, Sobek questions whether a five-month review period is "expeditious enough" to justify the hype surrounding the FDA's breakthrough therapy designation. However, if the administration approved ibrutinib even earlier than December, this would be hugely encouraging in shaping sentiment towards the new approval process.

From a commercial perspective, approval by the end of the year will be sufficient enough to boost revenue projections for ibrutinib and likely further accelerate growth in Pharmacyclics' share price (up 73.5 percent year-to-date). RBC Capital analyst Michael Yee suggests that launch in late 2013/early 2014 will provide ibrutinib more than a year's head-start on a competing therapy from Gilead Sciences and a two-year advantage over AbbVie's potential market entrant. Supporting the FDA’s strong supportive stance towards ibrutinib, Yee also suggests that Johnson & Johnson and Pharmacyclics' therapy is the potential market leader given both its efficacy and safety.

Goldman’s Singh now assigns a 100 percent success rate for ibrutinib in each of the indications for which it has been granted breakthrough designation and forecasts peak 2025 global sales of $6.7 billion for the franchise.

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