This blogger makes some good points.
He states, "The main reason is that approval doesn’t mean success." Couple that with the FDA not approving it and this is probably a good reason there isn't more excitement about this announcement.
NATURE NEWS BLOG
Cell-therapy conference feels (cautious) momentum
24 May 2012 | Posted by David Cyranoski | Category: Biology & Biotechnology
Last week, Columbia, Maryland-based Osiris Therapeutics boasted the “world’s first approved stem-cell drug”.
It isn’t, but the drug, Prochymal, used to treat graft-versus-host disease following transplant procedures, is still a rare regulatory success for a stem-cell therapy, and one might have expected some excitement about it at the World Stem Cells and Regenerative Medicine Congress in London this week.
It was rarely mentioned. They had other things to be excited, and worried, about. Amid a predictably buoyant mood for a meeting of industry hands, delegates discussed some successful regulatory approvals, large investment deals, a few cell-based therapeutics that are making revenue and the advancement of clinical trials over the last couple years — all of which, taken together, points to a ripening of the field, they say.
The conference covered not just stem cells, but all cellular therapies: anything in which biological cells are use for therapy. One of the meetings organizers, Chris Mason, chair of regenerative medicine bioprocessing at University College London, is keen to stamp out the distinction of stem cells. “I don’t like the word [stem cells],” he says. “They are all just living cells as therapies.”
Mason, who doubles as a partner in the investment and advisory firm Proteus Regenerative Medicine, says that growth has been strong across the industry. There are now 2,700 clinical trials in cellular therapies, 700 companies with mature technologies, and the 8 cell-based products approved in the United States and Europe now account for a more than US$1-billion industry, which he expects to grow to more than $3 billion by 2014. The lion’s share of that revenue is accounted for by three drugs, each with revenue of more than $200 million: the cancer drug, Provenge, and two skin grafts used for diabetic ulcers, Demagraft and Apligraf. The conference itself, in its seventh year, drew 350 people, its largest showing yet.
Hot topics were deals, like the $750 million that Dublin-based Shire paid for Dermagraft proprietor, Advance BioHealing in 2011 and Teva’s $6.8-billion purchase of Cephalon. A slew of others, having tentatively proven themselves effective, are entering phase III trials.
Representatives discussed the status of their pipelines relatively openly, largely because they are facing common enemies. One is the difficulty they face in getting health-care systems to agree to pay for the treatments once they are approved. The main target, however, are the regulatory agencies, many of which had representatives at the meeting. These agencies are alternately cajoled and condemned, with some stressing the importance of “getting in with the FDA [US Food and Drug Administration] early in the development process”, and others, usually behind the scenes, describing their own experiences as Kafkaesque attempts to navigate an endless maze.
The audience was hungry for any hints towards understanding the mysterious workings of the FDA. Geoff MacKay, president and chief executive of Organogenesis, whose gum-tissue-regenerating therapy was approved by the FDA in March, gave a blow-by-blow account of the process, which included three mock assessments the company carried out. He even included photos of the seating arrangement in the FDA assessment room.
Regulatory approvals are still rare. Tigenix’s ChondroCelect, approved in 2009, is still the only cell-based therapy on the market in Europe. So why wasn’t there more excitement about Prochymal’s approval? First of all, it wasn’t the first stem-cell therapy approved. South Korea has approved three over the past year.
Second, it had been rejected by the FDA last year and was approved by Canada, a smaller target and lesser coup, based on a subset analyses.
The main reason is that approval doesn’t mean success. Indeed, the company that brought Dermigraft to approval in 2001 went belly-up. Dean Tozer, senior vice-president of Advanced BioHealing, which “rescued” the therapy and made it profitable, says: “Approval is nice, but the question is whether it can be commercially viable.” Many are also withholding judgement on approved therapies in South Korea until they show profitability and success in the post-marketing research that the Korean FDA demands of them.
Antonio Lee from one of those Korean companies, Medipost, hoped that Osiris’s success in Canada, together with the recent spate of approvals in South Korea, might open up discussions on fast-track approvals in other countries, especially in the United States and Europe. No, he was told by Paula Salmikanga, the Finnish vice-chair of the European Medicines Agency Committee for Advanced Therapies. “The definitions and the requirements are all different,” she says.
Osiris shares rose 14%, but the Cellular Therapy Industry Index, which charts the top 30 companies in the field, was unaffected. Mason sees a bright side to that non-happening. He senses “a maturing of the investment environment” where one setback or one success no longer has a huge impact. “The market has settled over the last 18 months. It used to be people that wanted to make a quick buck. Now it’s serious investment people with long-term view.”
But it’s not clear there are enough of those investments, and aside from reimbursement debates and regulatory hardship, a pining for financial support filled the backdrop. As a representative of one large health-care investment company who did not want to be identified said: “I see VCs [venture capital companies] that say they have no money and I see pharmaceutical companies that say they don’t have any money. It’s a grim situation.”
