Anti Hunter Syndrome Drug Approved By FDA

Anti Hunter Syndrome Drug Approved By FDA

Hunter Syndrome, which usually becomes apparent in children one to three years of age, is a disease in which the person's body is defective in producing the chemical iduronate-2-sulfatase, which is needed to adequately breakdown complex sugars produced in the body. Symptoms include growth delay, joint stiffness, and coarsening of facial features. In severe cases, patients experience respiratory and cardiac problems, enlargement of the liver and spleen, neurological deficits, and death.


Hunter syndrome, or mucopolysaccharidosis II (MPS II), is a serious genetic disorder that primarily affects males. It interferes with the body's ability to break down and recycle specific mucopolysaccharides (mew-ko-pol-ee-sak-ah- rides), also known as glycosaminoglycans (gli-ko-sah-mee-no-gli-cans) or GAG. Hunter syndrome is one of several related lysosomal storage diseases.

Physical manifestations for some people with Hunter syndrome include distinct facial features, a large head, and an enlarged abdomen. People with Hunter syndrome may also experience hearing loss, thickening of the heart valves leading to a decline in cardiac function, obstructive airway disease, sleep apnea, and enlargement of the liver and spleen. Range of motion and mobility may also be affected. In some cases of Hunter syndrome, central nervous system involvement leads to developmental delays and nervous system problems. Not all people with Hunter syndrome are affected by the disease in exactly the same way, and the rate of symptom progression varies widely. However,Hunter syndrome is always severe, progressive, and life-limiting.

Recently,the Food and Drug Administration (FDA) today approved Elaprase (idursulfase), the first product for the treatment of Hunter syndrome (Mucopolysaccharidosis II, or MPS II), a rare inherited disease which can lead to premature death. Elaprase is a new molecular entity, which is an active ingredient never before marketed in the United States.

Elaprase was designated as an orphan product by FDA. Orphan products, such as Elaprase, are generally developed to treat rare diseases or conditions that affect fewer than 200,000 people in the U.S. The Orphan Drug Act provides a seven-year period of exclusive marketing to the first sponsor who obtains marketing approval for a designated orphan product. Hunter syndrome is diagnosed in approximately one out of 65,000 to 132,000 births.

"This is the first product that brings help to a very small group of seriously ill patients who have no other treatment option," said Dr. Steven Galson, Director, Center for Drug Evaluation and Research. "This approval is a good example of how the Orphan products program can benefit the public health with urgently needed products that would otherwise not be commercially available."

Elaprase was approved after a randomized, double-blind, placebo-controlled study of 96 patients with Hunter syndrome showed that the treated participants had an improved capacity to walk. At the end of the 53–week trial, patients who received Elaprase infusions experienced on average a 38-yard greater increase in the distance walked in six minutes compared to the patients on placebo.

The most serious adverse events reported during the trial were hypersensitivity reactions to Elaprase that could be life-threatening. They included respiratory distress, drop in blood pressure, and seizure. Other frequent, but less serious adverse events included fever, headache and joint pain.

Because of the potential for severe hypersensitivity reactions, appropriate medical support should be readily available when Elaprase is administered. Patients and their physicians are encouraged to participate in a voluntary Hunter Outcome Survey which has been established to monitor and evaluate the safety and effects of long-term treatment with Elaprase.

Elaprase is manufactured by Shire Human Genetic Therapies, Inc., in Cambridge, MA.

Comments

Post new comment

Similar

Obesity Drug Showing also Effective for Diabetics

Obesity Drug Showing also Effective for Diabetics The drug reduced body weight and improved control over blood glucose and blood fats among people with Type 2 diabetes, according to the study, published online by the British health journal The

High Doses of the Steroid Cortisone Weaken Bones and Cause Osteoporosis

High Doses of the Steroid Cortisone Weaken Bones and Cause Osteoporosis Cortisone is sometimes used as a drug to treat a variety of ailments. It can be administered intravenously or cutaneously. One of cortisone's effects on the body, and a potentially harmful side

New Nsaid Shown Effective for Psoriasis

New Nsaid Shown Effective for Psoriasis Psoriasis is an immune-mediated disease which affects the skin and joints. It commonly causes red scaly patches to appear on the skin. The scaly patches caused by psoriasis are often called psoriasis

Hip Replacement, Hip Resurfacing & Birmingham Hip at low cost.

Hip Replacement, Hip Resurfacing & Birmingham Hip at low cost Birmingham Hip Resurfacing technology, is a much advanced technical procedure than conventional total hip replacement. As a novel procedure, it demands an experienced surgeon who can thoroughly

Duloxetine (Cymbalta) Might be Choice for The Treatment of Diabetic Neuropathy

Duloxetine (Cymbalta) Might be Choice for The Treatment of Diabetic Neuropathy Diabetes is the leading cause of neuropathy in developed countries, and neuropathy is the most common complication and greatest source of morbidity and mortality in diabetes patients. It is estimated

Long Term Taking of ASPIRIN Also Might Be Great Benefit for Head and Neck Cancer

Long Term Taking Of ASPIRIN Also Might Be Great Benefit for Head and Neck Cancer Aspirin was the first discovered member of the class of drugs known asnon-steroidal anti-inflammatory drugs (NSAIDs),often used as an analgesic (against minor pains and aches), antipyretic (against

Anti Chronic Myeloid Leukemia (CML) or Acute Lymphocytic Leukemia (ALL) Drug

Anti Chronic Myeloid Leukemia (Cml)/Acute Lymphocytic Leukemia(All) Drug:Vertex, Chronic myeloid leukemia/Chronic myelogenous leukemia (CML) occurs in all age groups, but most commonly in the middle-aged and elderly. Its annual incidence is 1 to 2 per 100,000 people, and slightly