Out of the blue, on 19 April 200, came a press release from Genzyme, announcing that they were buying the rights to the Synpac product. under the deal it seemed that Genzyme paid $20 million to Synpac and Pharming paid $10 million to Genzyme. It looked like the Genzyme/Pharming joint venture would now commercialise ERT using the Synpac product ie one produced in fermenter vessels using chinese hamster ovary (CHO) cells.
What on earth was going on? What would happen to patients already receiving the rabbit enzyme? Why was this happening? Was the Synpac enzyme better?
Rumours abounded. The Duke/Synpac trial results had not yet been published however it was put about that the results (based on 3 patients) were better than those for the Rotterdam trial. As it turned out, this was not the case. However it ushered in a period of great uncertainty for patients.
It is true that enzyme production in rabbit milk was more difficult than originally anticipated. The rabbits did not produce as much enzyme as hoped and the Rotterdam trial showed that more enzyme was needed than originally thought too. The initial estimate that one rabbit would produce enough for one patient was now looking very optimistic indeed. However, they could be seen as a stop-gap until a larger milk producer - cows - could be brought on-stream. That did not seem to be in the game plan though.
And while all the above may have been true (though not necessarily the show-stopper it was all purported to be) it is certainly true that the move to CHO production suited Genzyme. They had a track record of enzyme production by that method and manufacturing-scale facilities already in use for their Gaucher product. This turn of events locked Pharming into the CHO method for the joint project with Genzyme. As the path to commercialisation became longer, the drain on Pharming's resources became too much for the small company and they went into receivership on 10 August 2001 . A potential long-term competitor to Genzyme was removed; Pharming slept with the fishes (as far as the Pompe project went anyway - they survived receivership and are still a going concern).
Of course, I am in no way suggesting that this is an outcome that Genzyme were actively working towards - it's just the way things turned out.
There was certainly no lack of commitment to the success of the joint venture, as evidenced by the recruitment of Paul Kaplan, who had a 10-year record in drug development, to lead it into the next phase.
I'm getting ahead of myself there though. All this was still to unfold. For now (and for the next few years) the IPA was involved in a battle to ensure that those on the Pharming trial continued to receive the Pharming enzyme, on which they were doing demonstrably well.
It was unsettling - things just didn't seem to be progressing to the timescale that patients would have liked. There was a suspicion that this might be due to the lack of competition.
Then, all of a sudden, there was competition again - in the form of Novazyme, a new company headed by John Crowley.
The notes of IPA meetings at the time show that