November 19, 2018Regulatory
October 22, 2018Regulatory
September 25, 2017Regulatory
July 10, 2017Regulatory
For our last Orphan Drug Designation Request, FDA told us that there could be a 6 month review time. We did contact the Division and were told about the major backlog.
June 12, 2017Regulatory
The FDA has requested that Endo Pharmaceuticals remove its opioid pain medication, reformulated Opana ER (oxymorphone hydrochloride), from the market. After careful consideration, the agency is seeking removal based on its concern that the benefits of the drug may no longer outweigh its risks. This is the first time the agency has taken steps to remove a currently marketed opioid pain medication from sale due to the public health consequences of abuse.
June 5, 2017Regulatory
Giant cell arteritis is a form of vasculitis, a group of disorders that results in inflammation of blood vessels. This inflammation causes the arteries to narrow or become irregular, impeding adequate blood flow. In giant cell arteritis, the vessels most involved are those of the head, especially the temporal arteries (located on each side of the head). For this reason, the disorder is sometimes called temporal arteritis. However, other blood vessels, including large ones like the aorta, can become inflamed in giant cell arteritis. Standard treatment involves high doses of corticosteroids that are tapered over time.
May 30, 2017Regulatory
EDITOR'S NOTE: THIS APPROVAL IS BEYOND HUGE AS WE CAN NOW START TREATING THE ROOT CAUSE OF DISEASES RATHER THAN THEIR ORGAN (e.g. Colon Cancer)
May 22, 2017Regulatory
Cystic fibrosis is a rare disease that affects about 30,000 people in the United States and affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery due to the movement of sufficient ions (chloride) and water in and out of the cells. People with the progressive disease have a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene that can't regulate the movement of ions and water, causing the secretions to become sticky and thick. The secretions build up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.
May 15, 2017Regulatory
An estimated 1 in every 2,500 babies in the U.S. is born with esophageal atresia. Babies with this condition cannot feed normally, and they require a feeding tube until surgery can be performed to attach the esophagus to the stomach. Most babies born with esophageal atresia also have a tracheoesophageal fistula, which also needs to be repaired surgically, since fluids from the esophagus can get into the airways and interfere with breathing.
May 1, 2017Regulatory
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency, is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease. CLN2 disease is a rare inherited disorder that primarily affects the nervous system. In the late infantile form of the disease, signs and symptoms typically begin between ages 2 and 4. The initial symptoms usually include language delay, recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia). Affected children also develop muscle twitches (myoclonus) and vision loss. CLN2 disease affects essential motor skills, such as sitting and walking. Individuals with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens. Batten disease is relatively rare, occurring in an estimated two to four of every 100,000 live births in the United States.