jandekkers schreef op 21 september 2017 23:10:
Peer reviewed validation for prophylaxis data. A driver to the positive FDA interaction, in our belief, was the recent announcement that the positive Phase 2 prophylaxis data were published in The Lancet (data originally presented
in November 2016). The randomized, placebo-controlled study showed significant reductions in the number of HAE attacks with 22 of 23 patients (95.7%) experiencing >50% reduction with twice weekly Ruconest. Additionally, while
Ruconest is currently only approved for the acute HAE setting, we have discussed how treating physicians are increasingly looking to tailor treatment to the individual. To this end, while specifics not available, when patients are not adequately controlled in the prophylaxis setting such as Cinryze, due to breakthrough attacks, physicians are free to use other options, and we believe Ruconest is one of these options (helping to broaden positive experience with the drug). The FDA is also holding a public meeting on September 25 to discuss the treatment landscape for HAE, which is importantly expected to include patient input. We believe this event should increase visibility and continue to support Ruconest's profile.
Total market size for HAE treatments from actual product sales is estimated to come close to $800 million per year in the US alone — $400 million for HAE acute attack treatment and $400 million for prophylactic HAE treatment with only a few competitors on the market.
When Pharming can capture a 50% market share (400 Million in Revenue), their potential (US) market cap should be adjusted based on ~21X multiple used in the industry.