EMA recommends conditional approval of bedaquiline for drug resistant TB with orphan drug status

EMA press statement

On 20 December 2013, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended granting a conditional marketing authorisation for bedaquiline (Sirturo) for use as part of a combination therapy for pulmonary multidrug-resistant tuberculosis in adult patients when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.

In the European Union, tuberculosis is an orphan indication and was estimated in 2011 to occur in 2.3 out of 10,000 people. Multidrug-resistant tuberculosis is defined as tuberculosis that is resistant to at least isoniazid and rifampicin, which are two major anti-tuberculosis medicines used in standard treatment. Approximately 450,000 cases of multidrug-resistant tuberculosis occur globally every year, which corresponds to approximately 5% of the world’s annual burden of tuberculosis.

In recent years, the burden of tuberculosis resistant to first-line therapy has increased rapidly in the absence of new treatment options. Multidrug-resistant tuberculosis is associated with a high mortality rate and poses a significant public-health threat as individuals infected with drug-resistant strains are unable to receive adequate treatment and can potentially spread their infection.

Sirturo is the first representative of a new class of medicines against mycobacteria. The Committee considered that Sirturo could contribute to responding to the high unmet medical need for new treatment options for pulmonary multidrug-resistant tuberculosis. It recommended granting conditional marketing authorisation because, although the data supplied by the applicant show that the medicine’s benefits outweigh its risks, the data are not yet comprehensive. Therefore, additional studies on the use of Sirturo should be conducted.

Sirturo is the third positive opinion recently granted by the CHMP for a medicine to be used in the treatment of multidrug-resistant tuberculosis, after the November 2013 recommendations for delaminid (Deltyba), also for a conditional approval, and Para-aminosalicylic acid Lucane.

Bedaquiline is manufactured by Janssen-Cilag and was designated as an orphan medicinal product.

Conditional approval allows the marketing authorisation of medicines that target areas of unmet medical need before comprehensive data sets are available, in order to speed up access to much needed new medicines.


EMA press statement. European Medicines Agency recommends approval of a new medicine for multidrug-resistant tuberculosis. (20 December 2013).

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