FDA says Hold Off on Zavesca (Miglustat) approval.

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.”

Currently Zavesca (Miglustat) is FDA approved for type 1 Gaucher disease when enzyme replacement therapy is not an option. The milestone that this really entails for the NPC community is the ability for patients who’s insurance companies will not approve for off label use because it is not FDA approved for NPC. That means right now there are probably half of the patients in the United States that are simply unable to give it a go for their loved one(s).

When dealing with the FDA you have three options generally that will take place after submitting information for FDA approval:

1. An Approval Letter
2. A Non-Approval Letter (Sorry don’t pass go)
3. A Complete Response Letter (Not yet, we want more info)

Actelion received number three, a Complete Response Letter which allows the pharmaceutical company to continue to explore and collect data which might more clearly show the positive impact that the drug has on the treatment of the disease (Niemann-Pick Type C).

Currently the European Union, South Korea, Brazil, Russia, Australia and Canada, Zavesca® is indicated for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease.

If you are a parent of a child/adult with Niemann-Pick Type C, we encourage you to write Actelion or the National Niemann Pick Disease Foundation regarding the findings and/or benefits you feel your loved one has experienced from this.   The power of people and especially a parent because you are the only one that will ever fight the hardest for your child.

Read the full press release here: http://www1.actelion.com/en/our-company/news-and-events/index.page?newsId=1392243

Today, there is no cure or effective treatment

Thank you for stopping by! Niemann-Pick Type C Disease is a every day reality for our two eldest sons’ Brisan and Parker including our whole family. Their prognosis captures the meaning of Niemann-Pick Disease Type C (NPC). It’s a genetic neurological disease that strikes children through accumulation of cholesterol and other excess fats in the cells of the liver, spleen, and brain. When brain cell function is blocked, the child loses coordination, stumbles, falls, and eventually will need a wheelchair, hospital bed, and other adaptive equipment. As the disease worsens, other devastating symptoms develop, including loss of the ability to speak and swallow, and seizures may occur. The health of children with NPC declines until ultimately, the disease claims his or her life. This disease affects roughly a 75 people in the US and around 500 worldwide (ever). The NPCF was formed to help create a entity that we could bring to communities that surround us the awareness needed to shed light on a disease that researchers are taking interest in. We felt after facing this reality that research has a different time line than parents yet they are making progress then why could we not help out in some shape, way or form?

Today, there is no cure or effective treatment for Niemann-Pick, but research is making progress. Tomorrow through research made possible by the financial support of generous contributors, these precious children can be saved and hopefully our Brisan and Parker! But they need your help now. This disease once solved or partially will provide a gateway into other storage disorders and it’s relation to Alzheimer’s, atherosclerosis, HIV/Aids, and stroke.

The other ugly truth that we face as their parents is the disease is always fatal at the time I write this. Can you imagine the brutal honesty of the this whole situation?  Pretty surreal at times… can someone pinch us? We were told at the appointment of the official diagnoses on 8/8/08 that we should take them home and love them the best way we can in the mean time.

We encourage you to cherish ever moment with your family and don’t take for granted the intuitions you may have. The NPCF will need your support to make a  bigger impact! If you have any ideas or collaborations please reach out to us.

Sincerely,

Michael & Jennifer Stults