by Robert Galbraith

FLORENCE, Oct 25 (APM) - GlaxoSmithKline decided to price gene therapy Strimvelis within a long-term strategy which favours sustainability of health systems but may not make a profit, according to the group's head of gene therapy development, Sven Kili.

A symposium to discuss the new treatment was held on Friday at The European Society of Gene and Cell Therapy's 2016 Congress in Florence.

Strimvelis, which was approved in Europe in May (APMMA 48052), is used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). A child's own stem cells are harvested and used to manufacture the therapy, which is then administered back to the patient to correct the genetic disorder, the symposium heard.

The therapy was developed in partnership with Italy's Fondazione Telethon and Milan's Ospedale San Raffaele (OSR), which is the only centre where it can be administered. It is manufactured by another GSK partner, Milan-based biotech MolMed.

Strimvelis has been priced at 594,000 euros for a single infusion. There have been suggestions that GSK will provide the treatment with a money-back guarantee, although this was not confirmed at the conference.

During the symposium, Kili stressed the difficulties in pricing new gene therapies. He made it clear that GSK wanted to go down a different path from that for the first approved gene therapy, uniQure/Chiesi's Glybera (alipogen tiparvovec) for lipoprotein lipase deficiency, which was priced at 1.1 million euros (APMMA 40620). The commercial viability of this therapy, which has only been used once, has been called into question.

Kili did not mention the therapy by name, saying only that "most people are aware that another therapy was priced differently". He stressed the difficulty of placing a value on Strimvelis, a treatment for very small children which may only be needed once. "How do you do that without bankrupting health systems?" Kili asked.

He said that the company talked to different regulators and reimbursement authorities to explore the ways which could be used to pay for these therapies. It was clear that they wanted to have access to new gene therapies - but not at any price.

"We thought that with other therapies to follow, we didn't want to set a precedent, " Kili said. "We see at as investment for the future. With this price it is not that we'll make a big profit, we may not make a profit on Strimvelis," he told the symposium.

He described the pricing strategy for Strimvelis as "responsible". He explained that it takes into account the costs but will allow the development process of other gene therapies to continue, suggesting that there will be more opportunities further down the line.

Earlier, Kili had noted that GSK's alliance with Fondazione Telethon and OSR, which was formed in 2010, foresees the development of seven gene therapies in total.

Alessandro Aiuti, clinical research coordinator for the Telethon/OSR unit TIGET, described how the partnership came about. "We had excellent science and clinical data, but we were stuck because we could not see how we would put it (Strimvelis) onto the market which led us to choose the partnership. We think this is a winning strategy," he told the symposium.

Jonathan Appleby, GSK's medicines development leader, also stressed the importance of the alliance. "I think it is because the clinical data were very clear at the beginning of the process, the expertise was in San Raffaele and MolMed brought its share (for manufacturing). It was a complex environment, but each party knew what it had to do," he said.

Another issue raised during the symposium was the need for patients to travel to Milan for treatment. Kili said this is necessary because only MolMed is authorised to manufacture Strimvelis.

"Strimvelis is currently available as a fresh formulation and so treatment is only available in Milan," he explained. "We made a conscious decision about this because we tried to have it approved as quickly as possible, but we recognise that it is difficult for families to travel."

Kili said an "extended shelf life version" of the therapy is being developed so that Strimvelis will eventually be available in other centres in Europe.

/rg/clg