MONT ST. HILAIRE, Quebec, Canada -- Axcan Pharma Inc. announced that the Gastrointestinal Advisory Committee of the U.S. Food and Drug Administration ("FDA") has agreed that Axcan's investigational drug, PHOTOFRIN PDT, demonstrated efficacy and safety in the ablation of High-Grade Dysplasia associated with Barrett's Esophagus. The announcement was made following a review by the Committee of clinical data on PHOTOFRIN (porfirmer sodium) with Photodynamic Therapy ("PDT") in this indication. Axcan received an approvable letter from the FDA in the first quarter of fiscal 2003 and PHOTOFRIN PDT was recently approved in Canada for the same indication.
"We are very pleased that the Advisory Committee overwhelmingly agreed with the efficacy and safety of PHOTOFRIN PDT in this indication," commented François Martin, Senior Vice President, Scientific Affairs of Axcan. "Although the Committee was not asked to vote on whether it recommended PHOTOFRIN for approval, we now expect that the FDA will give us a final response by the end of Axcan's fiscal year," he concluded.
The Advisory Committee reviewed efficacy and safety data from studies with PHOTOFRIN PDT. Axcan submitted the results of three clinical trials in which PHOTOFRIN PDT was used for mucosal ablation of High-Grade Dysplasia. PHOTOFRIN is an approved photosensitizing agent that is administered intravenously and is innocuous until activated by light. When target tissues are exposed to laser light at 630 nm, tissue necrosis occurs by at least two mechanisms, oxygen radical damage and anoxia due to thrombosis of capillaries.
@teaser:Study Results
@body:The Advisory Committee recommended the use of PHOTOFRIN PDT in patients with High-Grade Dysplasia in Barrets Esophagus who do not undergo esophagectomy. This proposed indication is based on the results obtained from a multicenter, randomized, controlled, partially blinded, 2-arm trial, in which 138 patients were randomized to PHOTOFRIN PDT + omeprazole and 70 patients to omeparazole only, as a control group. Patients were followed every 3 months until four consecutive endoscopic results were negative for High-Grade Dysplasia and then semi-annually until the last enrolled patient had completed at least 24 months of follow-up evaluation after randomization. The length of follow-up ranged from 2 to 3.6 years.
The primary efficacy endpoint, assessed after a minimum follow-up of 24 months, was the complete ablation of High-Grade Dysplasia. PHOTOFRIN PDT resulted in a Complete Response in 77% of treated patients, while omeprazole alone resulted in 39% (the difference between groups was significant, with p<0.0001).
Secondary efficacy endpoint analyses showed that 1) the most common type of Complete Response was CR1 in the PHOTOFRIN PDT group and CR3 in the omeprazole-only group; 2) the median duration of Complete Response was 987 days in the PHOTOFRIN PDT group and 98 days in the omeprazole-only group ( 3) the proportion of patients who progressed to cancer was about twice as high in the omeprazole-only group compared to PHOTOFRIN photodynamic therapy group (p=0.006). Additional analyses showed that patients who failed to achieve a Complete Response in either group of patients had an approximately ten-fold higher risk of progression to cancer than patients who achieved a Complete Response.
@teaser:About Barrett's Esophagus
@body:Barrett's Esophagus is a condition that results from prolonged heartburn in which the normal lining of the lower part of the esophagus is replaced, over time, by another type of lining normally present in the stomach. Barrett's Esophagus is clearly recognizable at endoscopy. Typically, Barrett's Esophagus develops during the process of healing after a chronic injury to the esophageal mucosa, such as the injury caused by the reflux of gastric juice in the esophagus. Continued reflux may cause dysplastic changes progressing from Low-Grade to High-Grade Dysplasia. Such dysplasia may lead to esophageal adenocarcinoma, a life-threatening condition.
It is estimated, in North America, that 25,000 to 35,000 people suffer from High-Grade Dysplasia associated with Barrett's Esophagus, and approximately 5,000 to 7,000 new patients in North America are diagnosed with this condition each year.
Axcan is a leading specialty pharmaceutical company involved in the field of gastroenterology. Axcan markets a broad line of prescription products sold for the treatment of symptoms in a number of gastrointestinal diseases and disorders such as inflammatory bowel disease, irritable bowel syndrome, cholestatic liver diseases and complications related to cystic fibrosis.
Information :
David W. Mims, Executive Vice President and Chief Operating Officer, Axcan Pharma Inc. Tel: (205) 991-8085 ext. 223 or Isabelle Adjahi, Director, Investor Relations, Axcan Pharma Inc. Tel: (450) 467-2600 ext. 2000