2012 yılında 8,7 milyon civarında yeni tüberküloz (TB) olgusunun geliştiği, 1,4 milyon kişinin bu sebeple yaşamını yitirdiği günümüzde, hastalığın küresel boyuttaki yükü büyümeye ve buna ilişkin zorluklar önemi nisürdürmeye devam ediyor. Çok ilaca dirençli gösteren tüberküloz (MDR-TB) ölümcül olduğu kadar tedavi edilmesi en zor tüberküloz türlerinden birisi olarak kabul ediliyor. Dünya genelinde yaklaşık 630 bin insanı etkisi altına alan hastalık dünyanın birçok bölgesinde yükselişini sürdürüyor.
Janssen, on yılı aşkın süredir gerçekleştirdiği klinik araştırmalar sonucu yeni ilacı Bedaquiline’i geliştirdi. Janssen’in yeni ilacı, uzun yıllardır ciddiyetini koruyan MDR-TB hastalığı için yeni bir etki mekanizması sunan ve bu alanda son 40 yılda geliştirilen ilk molekül olma özelliği taşıyor. Amerikan Gıda ve İlaç Dairesi’nin (FDA) Bedaquiline için vermiş olduğu hızlandırılmış onay, MDR-TB’ye karşı yürütülen savaşta atılmış büyük bir adım olma özelliği taşıyor. Janssen, Bedaquiline ile tüberküloz tedavi başarısını artırmayı ve bu sebeple ortaya çıkan istenmeyen sonuçları azaltmayı hedeflendiğini belirtiyor.
For Immediate Release: Dec. 31, 2012
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On Dec. 28, the U.S. Food and Drug Administration approved Sirturo (bedaquiline) as part of combination therapy to treat adults with multi-drug resistant pulmonary tuberculosis (TB) when other alternatives are not available. TB is an infection caused by Mycobacterium tuberculosis and is one of the world’s deadliest diseases. It is spread from person to person through the air and usually affects the lungs, but it can also affect other parts of the body such as the brain and kidneys. According to the Centers for Disease Control and Prevention, nearly 9 million people around the world and 10,528 people in the United States became sick with TB in 2011.
Multi-drug resistant TB occurs when M. tuberculosis becomes resistant to isonazid and rifampin, two powerful drugs most commonly used to treat TB. Sirturo is the first drug approved to treat multi-drug resistant TB and should be used in combination with other drugs used to treat TB. Sirturo works by inhibiting an enzyme needed by M. tuberculosis to replicate and spread throughout the body.
“Multi-drug resistant tuberculosis poses a serious health threat throughout the world, and Sirturo provides much-needed treatment for patients who have don’t have other therapeutic options available,” said Edward Cox, M.D., M.P.H, director of the Office of Antimicrobial Products in the FDA’s Center for Drug Evaluation and Research. “However, because the drug also carries some significant risks, doctors should make sure they use it appropriately and only in patients who don’t have other treatment options.”
Sirturo is being approved under the FDA’s accelerated approval program, which allows the agency to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug’s clinical benefit and safe use.
The FDA also granted Sirturo fast track designation, priority review and orphan-product designation. The drug demonstrated the potential to fill an unmet medical need, has the potential to provide safe and effective treatment where no satisfactory alternative therapy exists, and is intended to treat a rare disease, respectively.
Sirturo carries a Boxed Warning alerting patients and health care professionals that the drug can affect the heart’s electrical activity (QT prolongation), which could lead to an abnormal and potentially fatal heart rhythm. The Boxed Warning also notes deaths in patients treated with Sirturo. Nine patients who received Sirturo died compared with two patients who received placebo. Five of the deaths in the Sirturo group and all of the deaths in the placebo arm seemed to be related to tuberculosis, but no consistent reason for the deaths in the remaining Sirturo-treated patients could be identified.