CellPro Inc. received an approvable letter from the FDA regarding itsstem cell isolation system, which, when approved, will be the first toreach the U.S. market.
The Seattle company said late Tuesday the premarketing approvalapplication for the Ceprate SC stem cell concentration system isapprovable pending final resolution of labeling issues and otherquestions related to manufacturing and limited additional data.
Company officials said they already supplied the clinical data andaddressed other questions. The requirement for a GMP plantinspection, which hasn't been scheduled, appears to be the onlyhurdle.
CellPro stock (NASDAQ:CPRO) gained $1.75 Friday to close at$18.
Marc Ostro, a managing director at New York-based UBS Securities,still is targeting approval in the September time frame. He said asignificant point to the news is the FDA letter came in just twomonths.
Questions posed by the FDA at the advisory committee meeting had anegative slant, Ostro said. "A lot of people believed, in spite of the13-0 advisory panel vote [for approval], they were going to drag theirfeet on this. The fact they got the approvable letter in two monthsshould allay those fears."
In December 1994 the FDA sent CellPro a non-approvable letter forthe product, and in 1995 former European marketing partner CorangeLtd., of Hamilton, Bermuda, opted out of the deal. But CellProalways intended to market Ceprate SC itself in the U.S., and launch isnot expected to pose any problems once final clearance is granted,said Lee Parker, CellPro's director of investor relations.
Even with the delays CellPro won't have a U.S. competitor for atleast two years, Ostro said.
The system consists of a computerized base instrument, alreadyinstalled in 60 to 65 U.S. centers, and disposable, one-per-patient kitscontaining tubes, filters and a body of antibodies for injection into thebone marrow. Ceprate separates human stem cells from others cellsof the bone marrow or peripheral blood. The separated cells arereturned to the patient after chemotherapy or other marrow-destroying procedures to help restore bone marrow.
Parker said the two years of experience CellPro has in Europe, inaddition to the fact that systems are set up already in about one-quarter of the 250 bone marrow transplant centers in the U.S., shouldresult in a smooth U.S. launch. CellPro plans to target those centerswith a sales force of about 25, very similar to what it's doing inEurope.
Parker said more than 200 institutions in the U.S. and Europe areusing Ceprate as part of their own clinical studies.
So while the initial labeling might be for autologous bone marrowtransplantation, Ceprate likely will be used for a number ofapplications, such as procedures involving peripheral blood stemcells (PBSC) and allogeneic transplants. The science of PBSC wasnot developed when CellPro was running the trials on which thesubmission was based, but that procedure is becoming more popularand is expected to be used more than bone marrow transplantations.
The base system sells in Europe for about $20,000 and the disposablekits about $4,400 apiece. Revenues will be generated primarily fromthe sales of the kits. Sales in Europe for the year that ended in March1996 exceeded $6 million, Parker said, adding that in the U.S. thereare about 21,000 procedures per year done that could benefit usingCeprate.
CellPro is studying the device in a Phase III trial in multiple myelomapatients. The company is trying to demonstrate use of Ceprate toselect stem cells, while ridding tumor cells, will help deplete thetumor after the cells are reinserted to repopulate the bone marrow.Because of one-year follow-up requirements a filing in that indicationwouldn't be made until early 1998.
CellPro also plans to begin preliminary trials this year on two second-stage devices, one that extracts T cells and one that uses a panel ofbreast cancer antibodies to further extract breast cancer cells. n
-- Jim Shrine
(c) 1997 American Health Consultants. All rights reserved.