European agency is 'positive' about SRI cancer drug

Researchers at Southern Research Institute in Birmingham inched closer to global approval for their most recent cancer drug to achieve U.S. Food and Drug Administration approval when Bioenvision Inc. (NASDAQ: BIVN) announced late Thursday the European Medicines Agency has adopted a "positive opinion" on the marketing authorization application for the drug.

Bioenvision holds the European marketing rights for the SRI-developed clofarabine to be marketed as Evoltra, while Genzyme Corp. holds the North American rights under the name Clolar.

Clofarabine is indicated for the treatment of relapsed or refractory acute lymphoblastic leukemia in pediatric patients.

The European agency's Committee for Medicinal Products for Human Use issued the positive opinion about one month ahead of schedule, and John "Jack" Secrist III, vice president of SRI's Drug Discovery Division, says that leaves only a three-month negotiating negotiation of a marketing authorization expected to take about 90 days.

Evoltra has already been granted orphan drug designation, providing marketing exclusivity for 10 years in Europe following the marketing authorization.

Secrist says development and commercialization of clofarabine has taken more than two decades, so Thursday's announcement is exciting.

"We couldn't find anybody who was willing to give this drug a chance. We talked to Big Pharma, small companies, companies around the world, and a lot were interested but we didn't have any formal agreements until Bioenvision came on board in (the late 1990s)," Secrist says.

"I think it's great that we can now formally provide access to this drug to pediatric patients that really need it in Europe. Fortunately there aren't very many (patients) who don't respond well to first-line treatment, but for those who don't, this is an excellent additional treatment option."

Secrist, who says the drug spent seven years in limbo between patent approval and licensing negotiations, says the drug's promise seemed to resonate most strongly with parents of pediatric leukemia patients who lobbied the FDA for approval.

"What they wanted was more options. You go through everything, and there's nothing left, and it's your child...but this provides a very good option," Secrist says.

Clofarabine received FDA approval Dec. 28, 2004.

Secrist says SRI will receive a royalty stream based upon any sales that either Bioenvision or Genzyme generate from the compound, but he says those figures are confidential.

He did say he does not expect the revenue to be significant in the short term because of the "fortunately" small number of patients affected, but he says the drug is showing clinical promise with adult leukemia indications for which both companies are currently seeking regulatory approval.

"Evoltra is the first new drug for children with multiple relapsed or refractory leukemia to receive a positive opinion in Europe in more than a decade. It offers a genuine hope of response and disease free survival for this patient group," said Hugh Griffith, Bioenvision's chief operating officer, in a written statement.

Christopher Wood, the company's chairman and CEO, called Evoltra's introduction into the European market the "cornerstone of Bioenvision's commercial strategic plan."

According to the release, once the drug is formally approved via the Centralized Procedure, Evoltra can be marketed throughout all 25 member states of the European Union which has a population of about 450 million.