PhRMA: More than 400 rare disease treatments in development, pending approval
WASHINGTON — More than 400 drugs are in late-stage clinical development or awaiting Food and Drug Administration approval as treatments for rare diseases, according to a report by the drug industry lobby.
The Pharmaceutical Research and Manufacturers of America said a record 460 drugs were in late stages of the pipeline in a report timed to coincide with Rare Disease Day on Feb. 28. The rare or “orphan” diseases — defined by the government as those that affect fewer than 200,000 Americans — range from autoimmune disorders to cancer to blood disorders to genetic diseases. In total, there are 7,000 known rare diseases, about half of which affect children, and 80% of rare diseases affect fewer than 6,000 patients.
“Once you’ve talked to a desperate parent whose child is suffering or dying, you will be forever convinced of the need for medical innovation, including for terrible diseases that afflict even just a few,” PhRMA president and CEO John Castellani said. “Researchers at biopharmaceutical companies are working every day to bring hope to patients with limited options.”
Just a shout out to NORD ,Global Genes, Genetic Alliance and all of the wonderful organizations that are fighting for these treatments and for helping get more attention to Rare Diseases. As a Rare Mom, its a full time job finding the name of the disease, educating the medical community about it, and then getting the government to help us. This is an incredible community of intelligent, brave people.Great job moving the needle.
Fatty liver may impose diabetes risk
NEW YORK — Over the years, a fatty liver has become an indicator of obesity and insulin resistance among humans, but researchers have found that people with fatty livers are five times more likely to develop Type 2 diabetes than their healthier counterparts.
In a new study slated for publication in the Journal of Clinical Endocrinology & Metabolism, researchers examined 11,091 Koreans with fasting insulin concentration, a marker of insulin resistance, and diagnosed the subjects with a fatty liver via an abdominal ultrasound. At baseline, 27% of the population had been diagnosed with having fatty liver.
The researchers followed up after five years of the initial examination and found that regardless of baseline insulin concentration, patients with fatty livers had significantly more metabolic abnormalities, including higher glucose and triglyceride concentration and lower HDL ("good" cholesterol) levels. They concluded that patients with fatty livers were five times more likely to develop Type 2 diabetes.
"We believe that a diagnosis of fatty liver should raise an alarm for impending Type 2 diabetes," said Sun Kim of Stanford University and senior author of the study. "Our study shows that fatty liver, as diagnosed by ultrasound, strongly predicts the development of Type 2 diabetes, regardless of insulin concentration."
"Our study shows in a large population of relatively healthy individuals that identifying fatty liver by ultrasound predicts the development of Type 2 diabetes in five years," Kim added. "In addition, our findings reveal a complex relationship between baseline fatty liver and fasting insulin concentration."
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Protalix receives CRL for Gaucher disease treatment
KARMIEL, Israel — The Food and Drug Administration has declined to approve a biologic treatment for a rare genetic disorder by Israeli drug maker Protalix Biotherapeutics, Protalix said Friday.
The company said the FDA issued a complete response letter for its application for taliglucerase alfa, a treatment for Gaucher disease. The FDA issues a CRL when it has finished reviewing a regulatory application, but questions remain that preclude final approval.
The FDA raised questions about clinical trials, chemistry, manufacturing and controls, Protalix said. The drug maker said that when it submitted the application, the full data were not available, but that it would request a meeting with the FDA to clarify the path to regulatory approval “as soon as possible.”
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