By pursuing a particular disease pathway with a drug designed to treat a rare indication, X improves its shot at gaining an approval from a tougher set of regulators on both sides of the Atlantic. Then once the pharma has that initial approval it expands on the treatment label by adding a more common disease that is affected by the same pathway.
"A good example of this is Ilaris, which is for a rare set of autoimmune diseases called cryopyrin-associated periodic syndrome," "I think there are only 6,000 patients globally with this disease. But the mechanism, the pathway of this disease, is the same pathway that is prevalent in gout. And there are 3 million patients with acute gout. We have developed the drug in gout and we're awaiting FDA approval for this compound. This is a perfect example of how you would have multiple indications for the same drug and that's the way the drug would become--let's say, a blockbuster. Not that Ilaris will become a blockbuster but there will be multiple indications and multiple diseases through this fundamentally different approach."
FDA seems to be supportive of this approach. After all, platform drugs make sense. Once a product's safety is established in the first approved indication, the agency can generally be more secure that no serious unexpected safety issues will arise in a second, third, and fourth indication.
AVNRs 2nd and 3rd indications, MS pain and Alzheimer's agitation, should readout results first quarter 2014. With a typical three year Phase III, and one year to FDA approval, AVNR could be marketing some fairly good sized indications by 2018.
For MS central neuropathic pain, I think there will need to be two Phase 3 studies and I would give
those two Phase 3 studies at least five years from the end of Phase 2 to the results of the second Phase 3.
The results of the Phase 2 PRIME study are expected in early 2014, let's say March. Avanir should have an End of Phase 2 meeting with the FDA let's say, late June (which is optimistic) and optimistically would have a Phase 3 study designed and the first patient enrolled by December, 2014 with results optimistically by December,2016.
Then the second Phase 3 can begin, optimistically by , August, 2017 with results by August, 2019 and a New Drug Application filed and accepted by May, 2020. 10 months for normal FDA review gives a PDUFA date of March, 2021 and this is optimistic and assumes everything goes well and is only for central neuropathic MS pain, not agitation.
Agitation in dementia would probably take significantly longer (and I doubt dex/quin will get FDA approval for agitation in dementia.)