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Status Of Pharmaxis’ Orphan Drug Bronchitol In The US

January 31, 2013

On January 30, 2013, Pharmaxis receives an unanimous negative recommendation from the FDA Pulmonary-Allergy Drugs Advisory Committee (PADAC) Meeting for orphan drug Bronchitol (Mannitol) for the treatment of Cystic Fibrosis (CF).

PADAC voted on the following 3 questions about Bonchitol’s safety and efficacy for CF patients 6 years and older :

  1. Considering the totality of the data, is there substantial evidence of efficacy for DPM (Bronchitol) at a dose of 400 mg twice daily for improvement of pulmonary function in patients 6 years and older with cystic fibrosis? If not, what further efficacy data should be obtained?
  2. Is the safety profile for DPM (Bronchitol) for the maintenance treatment of patients with cystic fibrosis sufficient to support approval? If not, what further safety data should be obtained?
  3. Do the efficacy and safety data provide substantial evidence to support approval of DPM (Bronchitol) at a dose of 400 mg twice daily for the management of cystic fibrosis in patients aged 6 years and older to improve pulmonary function? If not, what further data should be obtained?

PADAC, composed of 14 voting members, deliberated over 7 hours and gave each of the 3 questions a negative result. The FDA will make its final decision on March 18, 2013. Pharmaxis says that “we are in a process and that opportunities remain to discuss the issues that were raised by the Committee before the FDA makes its final decision …..”.

Bronchitol has Orphan Drug Designation in the US, Europe, and Australia. It is approved for marketing in Australia and throughout the European Union. Bronchitol was discovered and developed in Australia and is currently on the list of Australia’s Pharmaceutical Benefits Scheme (PBS) for CF.

The following is the Internet Buzz on PADAC’s negative recommendation :

1)  Yahoo Finance Australia Video titled, “Pharmaxis drug development hits hurdle “

2)  The Australian January 31, 2013 article titled, “Pharmaxis takes a beating after US rejection”

3) January 30, 2013 article titled, “Panel rebuffs Pharmaxis’ Bronchitol for CF”

4)   The Brisbane Times January 31, 2013 article “Pharmaxis – the biotech that keeps bouncing back”.

There is good news for Pharmaxis, as the company signs a Financing Agreement with NovaQuest Pharma Opportunities Fund III, under which NovaQuest is to invest up to US$40 million to support continued development, manufacturing, and commercialization of Bronchitol for CF in the US and EU.

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