Target Health Blog

Regulatory

FDA Unveils Plan to Eliminate Orphan Designation Backlog

July 10, 2017

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Regulatory

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.

FDA Requests Removal of Opana ER for Risks Related to Abuse

June 12, 2017

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Regulatory

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.

First Drug Approved to Specifically Treat Giant Cell Arteritis

June 5, 2017

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Regulatory

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.

HUGE: FDA Approves First Cancer Treatment for Any Solid Tumor With a Specific Genetic Biomarker

May 30, 2017

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Regulatory

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)

FDA Expands Approved Use of Kalydeco to Treat Additional Mutations of Cystic Fibrosis

May 22, 2017

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Regulatory

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.

FDA Clears New Device to Treat Esophageal Birth Defect in Babies

May 15, 2017

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Regulatory

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.

FDA Approves First Treatment for a Form of Batten Disease

May 1, 2017

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Regulatory

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.

Drug Approved to Treat Tardive Dyskinesia

April 17, 2017

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Regulatory

Tardive dyskinesia is a neurological disorder characterized by repetitive involuntary movements, usually of the jaw, lips and tongue, such as grimacing, sticking out the tongue and smacking the lips. Some affected people also experience involuntary movement of the extremities or difficulty breathing. Tardive dyskinesia is a serious side effect sometimes seen in patients who have been treated with antipsychotic medications, especially the older medications, for long periods to treat chronic conditions, such as schizophrenia and bipolar disorder. Tardive dyskinesia can also occur in patients taking antipsychotic medications for depression and certain medications for gastrointestinal disorders and other conditions. It is unclear why some people who take these medications develop tardive dyskinesia yet others do not.

First Direct-to-Consumer Tests that Provide Genetic Risk Information

April 10, 2017

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Regulatory

The FDA has allowed marketing of 23andMe Personal Genome Service Genetic Health Risk (GHR) tests for 10 diseases or conditions. These are the first direct-to-consumer (DTC) tests authorized by the FDA that provide information on an individual’s genetic predisposition to certain medical diseases or conditions, which may help to make decisions about lifestyle choices or to inform discussions with a health care professional.

First Drug Approved for Primary Progressive MS

April 3, 2017

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Regulatory

On March 28, the FDA approved Ocrevus (ocrelizumab) to treat adult patients with relapsing forms of multiple sclerosis (MS) and primary progressive multiple sclerosis (PPMS). This is the first drug approved by the FDA for primary progressive multiple sclerosis (PPMS). The FDA granted this application breakthrough therapy designation, fast track designation, and priority review.

Helping to Speed Cures and Treatments to Patients

March 20, 2017

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Regulatory

The FDA is committed to helping deliver innovative, safe, and effective treatments and cures to the patients who need them as quickly as possible. To achieve this goal, FDA has implemented a variety of expedited review programs and are working to help shorten the development time before a product is even submitted for FDA review.

Mutual Recognition Promises New Framework for Pharmaceutical Inspections for United States and European Union

March 13, 2017

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Regulatory

The United States and the European Union (EU) completed an exchange of letters to amend the Pharmaceutical Annex to the 1998 U.S.-EU Mutual Recognition Agreement. Under this agreement, U.S. and EU regulators will be able to utilize each other's good manufacturing practice inspections of pharmaceutical manufacturing facilities. The amended agreement ?represents the culmination of nearly three years of U.S. FDA and EU cooperation as part of the Mutual Reliance Initiative and will allow the FDA and EU drug inspectors to rely upon information from drug inspections conducted within each other's borders. Ultimately, this will enable the FDA and EU to avoid the duplication of drug inspections, lower inspection costs and enable regulators to devote more resources to other parts of the world where there may be greater risk.

Approval of First Treatment for Nocturnal Frequent Urination to Overproduction of Urine

March 6, 2017

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Regulatory

Nocturia (wakening at night to urinate) is a symptom that can be caused by a wide variety of conditions, such as congestive heart failure, poorly controlled diabetes mellitus, medications, or diseases of the bladder or prostate.

FDA Approves Drug to Treat Duchenne Muscular Dystrophy

February 13, 2017

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Regulatory

Duchenne muscular dystrophy (DMD) is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

Newborn Screening System for 4 Rare Metabolic Disorders

February 6, 2017

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Regulatory

Target Health is very pleased that it worked closely with Protalix Biotherapeutics for the approval of Taliglucerase alpha for the treatment of Gauche disease, we and continue to work with Protalix in Fabry disease.

Elevated Levels of Belladonna in Certain Homeopathic Teething Products

January 30, 2017

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Regulatory

Homeopathic teething products have not been evaluated or approved by the FDA for safety or effectiveness. The agency is unaware of any proven health benefit of the products, which are labeled to relieve teething symptoms in children. In September 2016, the FDA warned against the use of these products after receiving adverse event reports.

FDA Approves Trulance for Chronic Idiopathic Constipation

January 23, 2017

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Regulatory

According to the National Institutes of Health, an estimated 42 million people are affected by constipation. Chronic Idiopathic Constipation (CIC) is a diagnosis given to those who experience persistent constipation and for whom there is no structural or biochemical explanation.

New NCI Drug Formulary Will Expedite Use of Agents In Clinical Trials

January 16, 2017

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Regulatory

The National Cancer Institute (NCI) has launched a new drug formulary (the “NCI Formulary”) that will enable investigators at NCI-designated Cancer Centers to have quicker access to approved and investigational agents for use in preclinical studies and cancer clinical trials. The NCI Formulary could ultimately translate into speeding the availability of more-effective treatment options to patients with cancer.

FDA Approves First Drug For Spinal Muscular Atrophy

January 9, 2017

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Regulatory

Spinal muscular atrophy (SMA), is a hereditary disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. There is wide variability in age of onset, symptoms and rate of progression.

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