FDA approves Pompe disease treatment
SILVER SPRING, Md. The Food and Drug Administration has approved a new treatment for a rare genetic disorder that causes muscle weakness, the agency said Tuesday.
The FDA approved Cambridge, Mass.-based Genzyme’s Lumizyme (alglucosidase alfa) for patients aged 8 and older with late-onset Pompe disease, which occurs in 1-in-40,000 to 300,000 births. Pompe disease causes heart and skeletal muscle weakness, leading to respiratory weakness and death from respiratory failure.
The disease results from the body’s genetic inability to make the enzyme alpha-glucosidase, also known as GAA, which is required for proper muscle functioning. The enzyme is needed to convert the sugar glycogen into energy; without the enzyme, glycogen builds up in the muscle cells, leading to weakness.
The drug is believed to work by replacing deficient GAA and reducing accumulated glycogen in the cells. Currently, the only other treatment available for Pompe disease is Myozyme (alglucosidase alfa), also made by Genzyme. Myozyme has lately been in short supply due to limited manufacturing capacity at Genzyme’s plants in Framingham and Allston Landing, Mass.
“Pompe disease is a devastating condition without the appropriate treatment,” said Julie Beitz, FDA Center for Drug Evaluation and Research Office of Drug Evaluation III director. “The approval of Lumizyme will provide an important treatment for patients diagnosed later in life with Pompe disease.”
The drug is being approved with a risk evaluation and mitigation strategy, or REMS, and will only be available through a restricted distribution system to ensure its use by the correct patient group. The drug will carry a boxed warning to alert patients and physicians to the risk of severe allergic and immune system reactions.
Dawn Professional names contest winner at industry event
CINCINNATI Procter & Gamble’s Dawn Professional brand has announced the winner of its “Make Your Restaurant Shine” contest at the National Restaurant Association Show in Chicago.
The Dawn Professional line — which provides comprehensive kitchen cleaning solutions to restaurants around the country — awarded Martha Bralkowski, the owner of Jordan’s Bar and Grill in Ridgway, Pa. The award was presented by chef Spike Mendelsohn on the World Culinary Stage, P&G said.
Mendelsohn presented Bralkowski with a $20,000 check for restaurant improvements. She also will receive a one-on-one expert consultation at her restaurant with Mendelsohn later this year. An experienced restaurateur, he will provide expert advice on how to best use the award, share feedback on Jordan’s menu and discuss how to use social media for success. Tips from this session will be featured at www.DawnCelebrations.com.
“Dawn Professional has always been a trusted partner of the restaurant industry, providing products and resources to help restaurants succeed. ‘Make Your Restaurant Shine’ is a way for us to help one deserving restaurant owner — Ms. Bralkowski — in a very personal way,” said Eric Hetrick, director, P&G Professional. “We hope this $20,000 award will help make Jordan’s Bar & Grill an even bigger part of the Ridgway, Penn., community.”
As the winner of the “Make Your Restaurant Shine” contest, Bralkowski also received a paid trip for four adults to the 2010 National Restaurant Association show now underway in Chicago. Contest applications were received online at www.DawnCelebrations.com through April 12.
FDA grants orphan drug designation for Friedreich’s ataxia treatment
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.”