Canada has a lot to celebrate for their current and future patients living with the “Childhood Alzheimer’s” or Niemann-Pick Type C (NPC). During the last full week of March 2010, the announcement came down that Health Canada [The Food Directorate, the Therapeutic Products Directorate and the Natural Health Products Directorate in the Health Products and Food Branch (HPFB)] approved the official use of Zavesca (Miglustat). Before now the only approved use was for Gaucher’s Disease Type 1. Continue Reading

On March 9, 2010 the FDA has requested additional preclinical and clinical information regarding Zavesca (Miglustat) before giving the ultimate approval that the Niemann-Pick Type C community was hoping for. Actelion stated that they will work diligently with the FDA to explore the best ways to address the points raised by the agency.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: “We remain committed to bringing an approved treatment to patients suffering from NP-C disease and in this spirit we will continue the dialogue with the FDA.” Continue Reading

We’re a few weeks behind in updating this information but some big steps have happened recently regarding the potential FDA approval for Zavesca. The US Advisory panel on Jan. 12th recommended approval!

This snippet is from the article on Reuters regarding the breaking news:

Doctors can prescribe Zavesca now for NP-C, but Actelion needs FDA clearance to market the drug specifically for that use. Patient advocates also said insurers are reluctant to pay for the drug for NP-C patients without the approval. The drug costs $159,000 a year per patient. Continue Reading