Shire submits regulatory approval application for Fabry disease treatment
CAMBRIDGE, Mass. Shire has submitted a regulatory approval application to the Food and Drug Administration for a biotech drug for treating a fatal genetic disease that affects approximately 10,000 people in the world, the company announced Tuesday.
The British drug maker is seeking approval for Replagal (agalsidase alfa), designed to treat Fabry disease. Fabry is a lysosomal storage disorder that causes deficiency of the enzyme alpha-galactosidase A, causing accumulation of the fatty substance Gb3 and resulting in symptoms such as cardiovascular and kidney dysfunction, pain, skin lesions and hearing and eye problems. The disease reduces the average patient’s lifespan by 15 to 20 years.
“We continue to deliver on our commitment to the Fabry community by filing a [biologics license application] to support long-term access to Replagal in the United States,” Shire Human Genetic Therapies president Sylvie Gregoire said. “We understand that this has been a difficult time for patients, and we remain committed to doing all we can to support the Fabry community during the supply shortage and for the long term.”
Replagal won approval in the European Union in 2001 and is available to Fabry patients in the United States under a limited FDA-approved treatment protocol.
In other news, Shire has also submitted approval applications in the United States, Canada and the European Union for velaglucerase alfa, a treatment for Gaucher disease, also a lysosomal disorder.
New report questions glucose self-monitoring among noninsulin-dependent Type 2 diabetes patients
NEW YORK There’s no proof that self monitoring of blood glucose by Type 2 diabetes patients not dependent on insulin produces any benefits, according to a recent report by a German healthcare analysis organization.
The Institute for Quality and Efficiency in Health Care also said in its report that it remained unclear whether blood tests had an advantage over urine tests. Blood glucose monitoring remains important for Type 1 and Type 2 patients who do inject insulin, the report noted.
The report by IQWiG — the organization bases its acronym on its German name — used six trials, but came to the conclusion that the data from the trials were not enough to draw any conclusions on the long-term benefit of glucose self-monitoring among noninsulin-dependent Type 2 diabetes patients.
Former CDC head assumes new role at Merck’s vaccine division
WHITEHOUSE STATION, N.J. The former head of the Centers for Disease Control and Prevention will soon start work as the new president of Merck & Co.’s vaccine division.
Merck announced Monday that it had appointed Julie Gerberding as president of Merck Vaccines, effective Jan. 25. Gerberding was director of the CDC from 2002 to 2009, having led the agency through more than 40 emergency response initiatives for health crises.
“Vaccines are a cornerstone of Merck’s commitment to health and wellness,” Merck chairman and CEO Richard Clark said. “We are delighted to welcome an expert of Dr. Gerberding’s caliber to Merck.”