The U.S. Food and Drug Administration today approved Defitelio (defibrotide sodium) to treat adults and children who develop hepatic veno-occlusive disease (VOD) with additional kidney or lung abnormalities after they receive a stem cell transplant from blood or bone marrow called hematopoietic stem cell transplantation (HSCT). This is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition.
HSCT is a procedure performed in some patients to treat certain blood or bone marrow cancers. Immediately before an HSCT procedure, a patient receives chemotherapy. Hepatic VOD can occur in patients who receive chemotherapy and HSCT. Hepatic VOD is a condition in which some of the veins in the liver become blocked, causing swelling and a decrease in blood flow inside the liver, which may lead to liver damage. In the most severe form of hepatic VOD, the patient may also develop failure of the kidneys and lungs. Fewer than 2 percent of patients develop severe hepatic VOD after HSCT, but as many as 80 percent of patients who develop severe hepatic VOD do not survive.
“The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.
The efficacy of Defitelio was investigated in 528 patients treated in three studies: two prospective clinical trials and an expanded access study. The patients enrolled in all three studies had a diagnosis of hepatic VOD with liver or kidney abnormalities after HSCT. The studies measured the percentage of patients who were still alive 100 days after HSCT (overall survival). In the three studies, 38 to 45 percent of patients treated with Defitelio were alive 100 days after HSCT. Based on published reports and analyses of patient-level data, the expected survival rates 100 days after HSCT would be 21 to 31 percent for patients with severe hepatic VOD who received only supportive care or interventions other than Defitelio.
The most common side effects of Defitelio include abnormally low blood pressure (hypotension), diarrhea, vomiting, nausea and nosebleeds (epistaxis). Serious potential side effects of Defitelio that were identified include bleeding (hemorrhage) and allergic reactions. Defitelio should not be used in patients who are having bleeding complications or who are taking blood thinners or other medicines that reduce the body’s ability to form clots.
The FDA granted the Defitelio application priority review status, which facilitates and expedites the development and review of certain drugs in light of their potential to benefit patients with serious or life-threatening conditions. Defitelio also received orphan drug designation, which provides incentives such as tax credits, user fee waivers and eligibility for exclusivity to assist and encourage the development of drugs for rare diseases.
Defitelio is marketed by Jazz Pharmaceuticals based in Palo Alto, California.
NAFDAC Raises Concern For Treatment Of Serious Pain
The fight against opioid excessive abuse has been on going for a while in U S and Nafdac says ”inadequate opioid for the treatment of serious pain and lack of access to narcotic drugs for the management of similar conditions have remained a major health concern especially people suffering from these health conditions.”
The Acting Director General of NAFDAC, Mrs. Yetunde Oni who expressed this concern at the presentation and dissemination of the 2015 Annual Report of the International Narcotics Control Board (INCB) at NAFDAC corporate headquarters in Abuja this month, said the Federal Ministry of Health and NAFDAC with the support of INCB have made tremendous progress toward meeting the country’s requirements of opioids.
She noted that under the National Drug Control Master Plan, provisions have been made to decentralize the distribution of narcotic drugs by the establishment of zonal drug distribution centres in the six geo-political zones to enhance access to these drugs for licit purposes.
Speaking further, Mrs. Oni said the Agency is conscious of the persistent and emerging challenges regarding drug problem in Nigeria and is working assiduously with relevant stakeholders, National Authorities and international organizations to address this challenge.
Particularly, she noted that the National Drug Control master plan developed under the auspices of EU-Funded NGAV16 project implemented by UNODC has provided a robust framework for drug control and given the National Authorities a window for closer collaboration and co-ordination.
This collaboration according to her is what the Agency has adopted in monitoring of legitimate trade in precursors, using the Pre-Export Notification Online (PEN Online), an initiative of INCB.
The Chairman of National Drug Law Enforcement Agency (NDLEA), Col. Muhammad Abdallah (Rtd) in his address said some cases of diversion of imported precursor chemicals have been detected by his agency leading to arrest of some culprits, stating that, the discovery and dismantling of 10 clandestine laboratories in Lagos and Anambra states by NDLEA between 2011-2015 attests to the illegal diversion of these products.
He regretted the upward trend in the use of Tramadol over the years which has led to significant seizures of the drug and arrest of suspects involved.
Reviewing the report, the Country Representative of United Nations Office on Drug and Crime UNODC, Ms. Cristina Albertin said the 2015 Annual Report focuses on the health and welfare of mankind: challenges and opportunities for international control of drugs.
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