Celgene’s Thalidomide Pharmion (thalidomide) has been granted full marketing authorisation by the European Medicines Agency (EMEA) for use in combination with melphalan and prednisone as a treatment for patients with newly-diagnosed multiple myeloma.

The approval was welcomed as “an important and positive milestone” by Celgene, which points out that multiple myeloma is the second most commonly-diagnosed blood cancer. More than 85,000 people in Europe are currently undergoing treatment for the condition, and 25,000 are forecast to die from it this year, it says.

“We are working diligently with local regulatory authorities to determine next steps for pricing, reimbursement and distribution plans for all European Union (EU) member states so that Thalidomide Pharmion will be available for eligible patients as quickly as possible,” said Aart Brouwer, President of Celgene International.

However, patient advocates have stressed the importance of making the drug available only through the Pregnancy Prevention Programme (PPP) being run by Celgene, which is now the only licence holder for the drug in the EU. The PPP monitors the distribution of every pill, requires women of childbearing age to follow a strict contraception programme and ensures that regular pregnancy testing is undertaken.

Groups representing patients with multiple myeloma and people born with deformities as a result of thalidomide have been working with the EMEA for more than five years to make sure that the drug is made available through the PPP only, said Freddie Astbury, president of Thalidomide UK.

The groups will meet with the regulatory authorities in each country to make sure they police the PPP “100%,” and implement an education programme to young doctors and pharmacists into “the dangers of thalidomide,” said Mr Astbury. Until the EU clearance yesterday, thalidomide had been distributed by many pharmaceutical companies “with little or no precautions to prevent a thalidomide child being born,” while many of the younger generation of doctors have not heard of the drug, know it under a different name or come from countries where it was never an issue, he added.

No manufacturer or distributor, apart from Celgene, may now supply thalidomide in any form across the EU. If they wish to do so, they must apply for a licence and put in place the procedures to adhere to the PPP.

The process of controlled access began in the USA with the System for Thalidomide Education and Prescribing Safety (STEPS) programme, which is similar to the PPP and has now been run successfully by Celgene for many years without a thalidomide baby being born, said Thalidomide UK’s chairman, Glenn Harrison, who added: “now having Europe on board, we intend to move on to other countries such as Brazil, where thalidomide damaged babies are still being born.”

Meantime, a rally was held in London earlier this month to mark the 50th anniversary of thalidomide’s introduction into the UK, by Distillers Biochemicals, as a treatment for morning sickness. The drug was subsequently withdrawn in 1961 after being linked to an increased number of babies born with deformities. Last Friday (April 18), survivors - who have received compensation from Distillers of around £18,000 a year but say this is not enough - gathered at the German Embassy in London to demand £3 billion compensation from Grunenthal, the company which developed the drug, and the German government. Grunenthal is currently in new compensation talks with German patients but points out that it has never sold the drug in the UK.