22 MARKET NEWS 'Breaktbrough'drug for cystic fibrosis approved in US and Canada

Patients with cystic fibrosis may breathe easier now that the US FDA and Canadian Health authorities have granted approval to Genentech's recombinant human DNase, Pulmozymef>. The drug, which breaks down DNA in bronchial secretions making them less viscous, is being heralded by some as a breakthrough. It is the first new treatment for cystic fibrosis in over 30 years and it took only 9 months from the time the application was first filed in the US to approval.

PI&1e m data aided approval In August, a US FDA advisory committee recom­

mended the approval of nebulised DNase 2.5mg once­daily for the treatment of cystic fibrosis symptoms in patients aged> 5 years with forced vital capacity (FVC) ~ 40 of predicted. This was based on a phase III trial involving 968 such patients. During the 6 months of treatment, the risk of developing a respiratory tract infection was reduced by 28% in patients receiving DNase 2.5mg once-daily and by 37% in patients receiving DNase 2.5mg twice-daily, compared with placebo treatment. The overall mean increase in forced expiratory volume in I second (FEV I) was 5.8 and 5.6 in the once- and twice-daily groups, respectively.

It is being stressed that DNAse is an adjunct to existing medications which should be continued as recommended.

Potential blockbuster There are approximately 30 000 patients with

cystic fibrosis in the US, a similar number in Europe, and around 3000 in Canada. Based on a treatment cost of around $US 10 000 per year, analysts predict that the new drug could have annual sales of around $500 million within 3 years.

DNase has already been approved in Austria, Sweden and New Zealand, and approval is expected soon in other major European countries. [See also Inpharma 907: 22,2 Oct 1993 and 900: 22, 14 Aug 1993] .."..,.,

8 Jan 1994INPHARMAe 0156-2703194/0919·000221$01.000 Adlalnternatlonal Limited 1994. All rlghb......".