Yesterday a Food and Drug Administration (FDA) advisory committee met to review a cystic fibrosis drug and provide the FDA with a recommendation of whether or not to approve the drug. After review, the FDA’s Pulmonary-Allergy Drugs Advisory Committee voted 14-0 against approval of Pharmaxis’ New Drug Application (NDA) for Bronchitol (mannitol) to improve pulmonary function in cystic fibrosis patients six years and older.
Additionally, the committee voted 11-3 in two separate votes stating that Pharmaxis had not provided sufficient evidence of efficacy and safety for the drug. Although the first study met its primary efficacy endpoint, the committee raised concerns regarding missing data. The committee was also concerned that the second study did not show significant effect on FEV1.
Committee members were especially concerned with the lack of evidence that the drug had significant effects in children. Some members noted that they would support approval of the drug in adult patients who were at least 18 years old.
Both studies had a high amount of treatment-related early discontinuations, raising safety concerns. The increased risk of hemoptysis in children was a major point. Some committee members thought that a smaller dose of the medication may be more beneficial.
“The Committee vote is disappointing, however, we are aware that these recommendations are not binding on the FDA and we will continue the process of working with the FDA to bring Bronchitol to patients in the US. It is important to remember 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 on 18 March 2013,” Alan Robertson, CEO of Pharmaxis stated.
Bronchitol is currently approved for marketing for patients six years and older in Australia and for patients 18 years and older throughout the European Union.
Separately than the decision by the panel, NovaQuest Pharma Opportunities Fund III LP has agreed to invest up to $40 million to support development of Bronchitol in the EU and the US. Under the terms of the agreement, Pharmaxis will receive $20 million initially; however NovaQuest is eligible for undisclosed payments that will be based on potential product sales.
“Pharmaxis explored a range of options to leverage its Bronchitol asset. The NovaQuest investment was selected because it is tailored to the expected growth of the business, it offers flexibility in the total investment amount, the investment returns to NovaQuest are based solely on the sales of Bronchitol, Pharmaxis retains the vast majority of expected sales revenue over the term of the agreement, and the strategic and operational control of the Bronchitol business remains with the Company,” Robertson said in a statement.
Source: Pharmaxis Ltd
Last Updated: 1/31/13; 12:15PM EST