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FDA grants orphan drug designation for Friedreich’s ataxia treatment

BY Alaric DeArment

WALTHAM, Mass. The Food and Drug Administration has granted special designation to encourage the development of a drug made by Repligen for treating a rare degenerative disease, Repligen said Monday.

Repligen said the FDA’s Office of Orphan Products Development granted orphan drug designation to the drug RG2833, a treatment for Friedreich’s ataxia. The FDA grants orphan drug designation to treatments for rare diseases and grants incentives such as a seven-year market exclusivity period, compared with the usual five years granted to new drugs.

Friedreich’s ataxia is a genetic illness affecting around 15,000 people worldwide that results from a gene defect causing insufficient production of the protein frataxin, leading to degeneration of nerves controlling muscle movements in the arms and legs and nerve tissue in the spinal cord. Symptoms of the disease typically emerge between the ages of 5 and 15 years and lead to severe disability, incapacitation and loss of life in early adulthood.

“We are very pleased to receive orphan drug designation for our Friedreich’s ataxia program,” Repligen CEO Walter Herlihy said. “RG2833 is the first compound that targets activation of the defective gene responsible for Friedreich’s ataxia. If this approach is successful, it has the potential to significantly impact patients’ lives.”

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Boehringer Ingelheim’s cancer drug may be ‘next generation’ treatment

BY Alaric DeArment

RIDGEFIELD, Conn. Data from a late-stage clinical trial of an investigational drug by Boehringer Ingelheim could show that the drug is a “next generation” treatment for cancers of the lungs, head and neck, BI said Friday.

The German drug maker plans to present results of a phase 3 trial of BIBW 2992 at the annual meeting of the American Society of Clinical Oncology in Chicago, which will take place between June 4 and 8.

BIBW 2992 is a pharmaceutical drug that targets the epidermal growth factor receptor and human epidermal receptor 2, also known as EGFR and HER2. Both are proteins that are known to promote the growth of cancer cells. BI said the drug shrank tumors in 22% of head and neck cancer patients, compared with 13% among those taking Eli Lilly’s and Bristol-Myers Squibb’s Erbitux (cetuximab).

The company also reported that the drug showed “significant” ability to fight tumors in patients with non-small cell lung cancer, and that 61% of patients with EGFR mutations had significant tumor shrinkage.

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Americans need more faith… in fiber

BY Michael Johnsen

WHAT IT MEANS AND WHY IT’S IMPORTANT Not eating enough fiber doesn’t kill you. But the disease states that result from a poor fiber diet do. Not a believer? Then you too may be a victim of IDS (irrational disbelief syndrome), the tongue-in-cheek faux condition that Fiber One touts as being the No. 1 cause behind America’s lack of fiber faith.

(THE NEWS: Lack of fiber in Americans’ diet caused by negative perception, Mintel finds. For the full story, click here)

The IDS condition may have been created out the mind of a General Mills marketer, but the lack of fiber in the typical American’s diet is real. As are the disease states that fiber deficiency can trigger such disease states as cardiovascular disease, colorectal and esophageal cancer, obesity, atherosclerosis, and coronary heart disease. A number of other less severe conditions is also correlated with a low fiber intake — elevated cholesterol levels, hemorrhoids, constipation, diarrhea and flatulence.

The real conclusion you can draw from this study is that Americans don’t know what they don’t know — fiber doesn’t taste good; only people suffering from digestive issues need to supplement their diets with fiber; all fibers are created equal. False, false and false. And that spells opportunity for a whole host of manufacturers in moving the needle around fiber education.

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