Replies to post #97502 on Biotech Values

[drbio45]

The response rate for Cinryze in acute is over 93%. Berinert (CSL) data in acute is quite good as well. Rhucin isn't going to work 100% of the time, so you must be splitting hairs or making it up as you go along.

Does CSL work in laryngeal and abdominal HAE? Pharmings does.

CSL product is pooled plasma isn't it -- they don't like the viral contam risk

In the U.S. Rhucin will have to break CSL's Orphan status to get on the market at all. And even then, I'd expect Rhucin US sales to be de minimis.

Of course, I am not privy to any information under a CDA so I haven't seen the FDA minutes, but I have always been told by all management that the FDA views human recombinant a separate drug to pooled plasma, they both have separate BLA filings,



There would be 3 companies splitting the smaller acute market (CSL, Dyax, Pharming) and Rhucin isn't sufficiently differentiated.

I think dyax is dead if Rhucin gets approved here. The data is better and dyax had cases of Anaphylactic Shock.


In Europe not only are C1's available but also Firazyr which is a pretty good drug for acute yet only sold $6M worth in 2009. I'd expect Rhucin sales to be less than Firazyr.


At investigator meetings no one wanted to use pooled plasma, in EU there is a larger concern of viral contamination compared to here. they have had more problems with viral contimination.
Firazyr causes a bee-sting like reaction. docs know it missed the primary endpoint in the randomized trial and Shire is having difficulty even recruiting patients.

[genisi]

In Europe not only are C1's available but also Firazyr which is a pretty good drug for acute yet only sold $6M worth in 2009.

Shire Reports Positive Phase III Icatibant Data to Address Nonapprovable Status in U.S.

http://www.genengnews.com/gen-news-highlights/shire-reports-positive-phase-iii-icatibant-data-to-address-nonapprovable-status-in-u-s/81244313/

Fast-3 study data will be submitted to FDA in early 2011.

Shire reports that data from an FDA-requested Phase III study with Firazyr® (icatibant) has confirmed that the drug significantly speeds time to relief from symptoms associated with acute attacks of hereditary angioedema (HAE). The Fast-3 study was carried out to support Shire's previously filed NDA for Firazyr in the U.S., which FDA deemed not approvable back in 2008. The firm says the Fast-3 trial data means it should now be able to file a complete response to the FDA’s nonapprovable letter during early 2011.

“These very positive and clinically meaningful results represent a significant milestone in the U.S. clinical development program for Firazyr,” notes Sylvie Gregoire, president of Shire HGT. Firazyr is currently approved in 37 countries, including the EU, for the symptomatic treatment of acute attacks of HAE in adults with a C1-INH deficiency.

The Fast-3 study involved 67 sites in 9 countries, and included 88 patients with moderate to very severe cutaneous and/or abdominal symptoms associated with HAE. Results showed that in comparison with placebo, treatment using subcutaneous Firazyr resulted in significantly faster time to onset of relief from combined symptoms, and also much faster onset of relief from the patient’s primary symptom. Median time to onset of composite symptom relief (measured as a 50% reduction in composite symptom score assessed by the patient) was 2 hours for Firazyr-treated patients, and 19.8 hours for those receiving placebo. Median time to onset of relief from the primary symptom (a 30% reduction in symptom score) was 1.5 hours for the Firazyr cohort, compared with 18.5 hours for the placebo-treated cohort.

Icatibant is a specific bradykinin B2 receptor antagonist administered by subcutaneous injection. Shire recently submitted data from the Phase IIIb EASSI study to the European Medicines Agency, to support its submission for a label amendment that would allow patients to self administer icatibant when they suffer an HAE attack.

Firazyr achieved worldwide sales of $3 million in the third quarter of 2010, up 71% on the equivalent 2009 period at constant exchange rates.