First oral drug for a deadly disease
This is a new orally available drug for one of the most poorly treated afflictions in neurology. "If there were ever a disease
with an urgent unmet medical need, it is MS," says Les Funtleyder, health care strategist at Miller Tabak.
Originally derived from a fungus, Isaria sinclairii, fingolimod (FTY720) uses a new mechanism of action involving a so-called S1PR agonist to bind selectively to circulating lymphocytes
and keep them from leaving the lymph nodes. This results in fewer activated T-cells in the bloodstream and central nervous
"All of the other MS drugs are injectibles, so the fact that it's an oral therapy is a big plus," says Funtleyder, noting
that many treatments, including interferons, also have debilitating side effects. "If it is approved, just proving non-inferiority
to other treatments will enable it to do well commercially."
The current Phase III trial is scheduled to end in 2013.
Will denosumab get a break?
A fully human monoclonal antibody that prevents the RANK ligand from binding to its receptor, denosumab inhibits the differentiation of osteoclasts, which break down bone, thus preventing the bone resorption associated with osteoporosis.
The drug is likely to win approval. The question is whether payers will spring for a premium over traditional therapy—and
especially the bisphosphonates, some of which are available as generics. Denosumab is a once or twice-yearly injection, which
has obvious compliance advantages. But the challenge will be to get payers to spring for a premium over bisphosphonates.
Amgen released Phase III datra this fall showing that denosumab outperformed Fosamax. Will it be enough? Or will Amgen have
to wait until the drug picks up a cancer indication to realize its potential?
Still waiting for prasugel
If ever a drug seemed to inspire FDA to sit on its hands, Effient (prasugrel) is it. An outside observer might be forgiven for assuming that FDA has most to gain by indecision.
Prasugrel promises to be a more efficacious alternative to Plavix, which will go generic in 2011, and to pose uncalculated
dangers, if doctors try to use it in populations too prone to bleeding. The potential for adverse effects, even with a strongly
worded warning, is high. And the drug will be pricey for payers.
"The issue for FDA is whether prasugrel is meaningfully better than Plavix," says Les Funtleyder. "All the recent drug withdrawals
have made them much more gun shy."
The drug may make it. "But with two FDA delays, its chances are not good," Funtleyder says.