PHARMAXIS JUMPS 70% ON BRONCHITOL EUROPEAN APPROVAL
October 25th 2011 01:48
Monday October 24, 2011
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PHARMAXIS
Pharmaxis jumped 70.2 percent to $1.60 following European approval of Bronchitol for cystic fibrosis late on Friday October 21, 2011.
The European Medicines Agency’s Committee for Medicinal Products for Human Use overturned its previous refusal to grant marketing authorization of Bronchitol for cystic fibrosis as an add-on therapy to best standard of care (BD: May 25, Jun 27, 2011).
The Committee restricted Bronchitol to adult cystic fibrosis patients and Pharmaxis chief executive officer Dr Alan Robertson told Biotech Daily that the adult market was 66 percent of the total number of cystic fibrosis patients in Europe.
Dr Robertson said the company was required to conduct a further paediatric trial for approval for patients aged six to 17 years.
In a telephone conference today, Dr Robertson said the additional trial would be of much shorter duration that the pivotal trial, taking “one to two months”, cost about $1.5 million to $2 million, but he would not be drawn on how long it might take to complete.
Dr Robertson said the company had accumulated data on children in trials to date and was confident that the drug would be approved.
“We’ll be filing as soon as we can for six years and over,” Dr Robertson said.
Dr Robertson said the European paediatric trial and finalizing European approval was in parallel with filing the new drug application for Bronchitol for cystic fibrosis to the US Food and Drug Administration, which was expected to be done by April 2012, with approval 10 months after filing, if the process went smoothly.
Dr Robertson said that final European marketing approval should be completed in January with stocks on the shelves by April and Bronchitol was the first new drug approved for cystic fibrosis in 15 years.
He said Pharmaxis would begin the roll-out in Germany which had 100 cystic fibrosis centres and the UK with 50 centres and then cover all 27 European Union countries.
Pharmaxis chief operating officer Gary Phillips told the telephone conference that Bronchitol would be priced at or “below where Pulmozyne was priced” at $12,000 per patient per year and plans to gain reimbursement were underway with each country having different processes.
Dr Robertson said the difference between the eight percent benefit figure generally used by his company to describe the phase III trial results and the two to three percent benefit described by the European Committee for Medicinal Products for Human Use was caused by the Committee using a different endpoint.
He said Pharmaxis found an eight percent benefit using the measure of forced expiration volume of air over one second (FEV-1) comparing patients tested before and after the use of Bronchitol, consistent with the FDA protocol, but the Committee compared the post-Bronchitol result to a “percentage predicted” basis.
Dr Robertson said that even using the higher standard the review by the Committee decided that the benefit outweighed the risks.
The Committee said that two adverse events in the trial were a narrowing of the airways in the lung and the coughing-up of blood.
Dr Robertson said both had been referred to in the trial results, as broncho-constriction and haemoptysis, respectively.
He said haemoptysis was a symptom of cystic fibrosis and there was no difference between patients receiving 400mg Bronchitol (mannitol) and the 50mg mannitol control.
Dr Robertson told Biotech Daily that there were no current plans for a capital raising saying there were a number of different ways of addressing the company’s balance sheet which has three quarters of cash at the current burn rate.
Pharmaxis closed up 47 cents or 50 percent at $1.41 with 14.8 million shares traded.
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