Ebb & Flow
Investors voted with their feet after learning that they will have to wait even longer for regulatory clarity over competing compounds for Fabry disease from Genzyme (GENZ) and Transkaryotic (TKTX).
On Friday, the FDA postponed this week's meetings of the Endocrinologic and Metabolic Drugs Advisory Committee to review GENZ's Fabrazyme agalsidase beta and Replagal agalsidase alfa from TKTX. Both therapeutics are marketed in Europe, but the BLAs have been at the FDA for more than two years.
Even though the agency said the meeting will be held this year, investors dumped TKTX, which shed $2.69 to $30.31 on 4.5 million shares on Friday, putting it down $4.10 (12%) on the week. GENZ lost $0.34 to $21.42 on 7.7 million shares on the day.
The agency cited "administrative complications" for the delay, which look to be disagreements about the composition of the panel (see Product Development, A11).
In fact, GENZ held up well despite issuing another profit warning. The stock was down only $0.36 on the week after advancing $1.55 to $19.95 on 11.9 million shares on Monday's news that it lowered third quarter and 2002 full year EPS guidance to $0.26-$0.28 from $0.31-$0.33, and to $1.08-$1.11 from $1.18-$1.23, respectively.
The guidance reflected lower-than-expected end-user growth of Renagel sevelamer to treat end-stage renal disease, and continued reductions in Renagel wholesaler inventories. GENZ now expects third quarter revenues of $36-$40 million and full year sales of $155-$165 million, down 18-26% from June's guidance of $200-$210 million. Going into the year, GENZ had hoped to sell $177 million of the phosphate binder (see Product Development, A11).
Monday's uptick may have been helped by a regulatory slowdown for Shire (LSE:SHP; SHPGY), which is developing a competing phosphate binder (see below), and by news that the FDA accepted for filing the BLA for Aldurazyme laronidase to treat mucopolysaccharidosis I (MPS I), which is under development with BioMarin (BMRN). The FDA also granted priority review status to the enzyme replacement therapy, which means the agency will respond to the filing within six months of the July 29 filing date. The product is under review in Europe. BMRN popped $1.35 (31%) to $5.66