Cath Lab Digest

News

New Treatment for Late-Onset Pompe Disease
The FDA approved alglucosidase alfa (Lumizyme) for patients ≥8 years of age with late-onset, noninfantile Pompe disease, a rare genetic disorder. Alglucosidase alfa, marketed by Genzyme Corporation, is believed to work by replacing the deficient acid alpha-glucosidase necessary for proper muscle functioning, thereby reducing the accumulated glycogen in heart and skeletal muscle cells.

Alglucosidase alfa is being approved with a Risk Evaluation and Mitigation Strategy. It will only be available through a restricted distribution system called the Lumizyme ACE (Alglucosidase Alfa Control and Education) Program to ensure that it is used by the correct patient group. Alglucosidase alfa will carry a Boxed Warning because of the risk of anaphylaxis, severe allergic reactions, and immune-mediated reactions. The approval of alglucosidase alfa will ensure that treatment is available for all US adult Pompe patients in need of treatment.

The safety and efficacy of alglucosidase alfa are based on a clinical study in 90 patients, 10 to 70 years of age, with late-onset Pompe disease. The safety and effectiveness have not been evaluated in patients with infantile-onset Pompe disease or in patients ≤8 years of age with late-onset disease. The FDA states that these patients should be treated with Myozyme, rather than Lumizyme.
Recommendations Revised for Rotavirus Vaccines

The FDA revised its recommendations for rotavirus vaccines for the prevention of the disease in infants and has determined that it is appropriate for clinicians and healthcare professionals to resume the use of Rotarix and to continue the use of RotaTeq.

The agency reached its decision based on a careful evaluation of information from laboratory results from the manufacturers and the FDA’s own laboratories, a thorough review of the scientific literature, and input from scientific and public health experts, including members of the FDA’s Vaccines and Related Biological Products Advisory Committee.

According to the FDA, both vaccines have strong safety records, including clinical trials involving tens of thousands of patients as well as clinical experience with millions of vaccine recipients.

H1N1 Influenza Virus Test Cleared by FDA
The FDA cleared the Simplexa Influenza A H1N1, a test for the 2009 H1N1 influenza virus in patients with signs and symptoms of respiratory infection. Until this clearance, tests for 2009 H1N1 influenza were only available through an Emergency Use Authorization, which allows the FDA, based on the evaluation of available data, to authorize the use of unapproved or uncleared medical products or unapproved or uncleared uses of approved or cleared medical products, during the time a declaration of emergency is in effect.

Using specimens from nasal swabs or nasal aspirates, the Simplexa Influenza A H1N1 (2009) test simultaneously amplifies and detects 2 regions of the influenza virus genome and an internal control. A positive result indicates that the patient is infected with the 2009 H1N1 influenza virus, but the test does not indicate the stage of infection. The Simplexa Influenza A H1N1 (2009) test is manufactured by Focus Diagnostics Inc.

Injectable Osteoporosis Treatment for Postmenopausal Women
The FDA approved denosumab (Prolia), an injectable treatment for postmenopausal women with osteoporosis who are at high risk for fractures. Denosumab is manufactured by Amgen Inc and works to decrease the destruction of bone and increase bone mass and strength. An injection of denosumab is recommended once every 6 months.