Treatment Options for NPC

Health Canada Approves Zavesca for Niemann-Pick Type C
First Authorized Treatment in Canada

Dateline 03/24/2010:

Health Canada recently announced its approval of the drug Zavesca (miglustat) as the first authorized treatment for neurological symptoms of Niemann-Pick Disease Type C (NPC).  Zavesca is not a cure for NPC, but it has shown promise in treating symptoms related to NPC and in slowing the progression of the disease for some patients.

In addition to Canada, Zavesca has been approved for use in NPC in the European Union, South Korea, Brazil, Russia and Australia.  (Currently, in the United States, Zavesca can only be prescribed "off-label" for use in NPC -- see related FDA story below.)

Produced by Actelion Pharmaceuticals, Zavesca is also used for treatment of patients with Type 1 Gaucher Disease, another lysosomal storage disease.

To read the CCNNPDF's press release regarding Canada's approval of Zavesca, click here

To read the press release from Actelion, click here.

[Mar 24, 2010 mem ~ UPDATED 06/27/2014]

Niemann-Pick Disease Type C Information (Canada Only) is a site supported by Actelion where you can access comprehensive information about the symptoms, diagnosis and management of Niemann-Pick type C disease.  The site consists of a patient sections and a healthcare professional sections, so please choose the relevant option to enter the site.

Please note: This site is not intended for US residents!

Additional NPC Clinical Trials

National Institues of Health ~ Cyclodextrin Clinical Trial

National Institutes of Health ~ Histone Deacetylase Inhibitors (HDACi) Clinical Trial

[Jun 27th, 2014 ~ blg]

ALERT:   Zavesca© (Miglustat) marketed by Actelion Pharmaceuticals, Inc.

The National Niemann-Pick Disease Foundation has been alerted by the Niemann-Pick Disease Group in the United Kingdom, as to the existence of another pharmaceutical product called, “Zavesca”, that is causing some confusion within our Niemann-Pick Disease Type C community, families and patients throughout the world. 

The product, whose packaging is similar to that of the genuine product, is developed and marketed by Getz Pharma, a company based in the country of Pakistan.  This product can be easily and cheaply purchased on-line, whereas the genuine Zavesca© (Miglustat) marketed by Actelion Pharmaceuticals, will only be prescribed through your Medical Specialist, Physician and/or Consultant for the treatment of Niemann-Pick Disease Type C (or Gaucher’s Disease).  

The Getz product contains an anti-depressant and is not indicated for use in patients with NP-C; taking this product without advice from your doctor could cause adverse effects or serious harm. As always, we strongly recommend that you seek medical advice before beginning and/or changing any therapy or treatment for your family member diagnosed with NPD. 

For more information about Zavesca as a treatment option for Niemann-Pick Disease Type C, please follow this web link:

Please feel free to contact us at the NNPDF Central Offices if you have any questions, or if you require any further information. 

Thank you,


*Please note: Zavesca has NOT been approved by the U.S. Food and Drug Administration as a treatment for Niemann-Pick Disease Type C patients in the United States.


Nadine M. HillExecutive Director

National Niemann-Pick Disease Foundation, Inc.

[Dec 14, 2012 nmh] 

Actelion Receives U.S. FDA "Complete Response Letter" for Zavesca (miglustat)
for the Treatment of Niemann-Pick Type C Disease in the United States

ALLSCHWIL/BASEL, SWITZERLAND - 09 March 2010 - Actelion Ltd (SIX: ATLN) announced today that the company has received a complete response letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for Zavesca® (miglustat) for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare, neurodegenerative genetic disorder.

The FDA has requested additional preclinical and clinical information. Actelion 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."

Read Actelion's press release here:

Read the NNPDF's press release here:  Zavesca -- FDA "Complete Response" Letter

Dear Families of the NNPDF:

It is with deep regret that I pass along word that the United States FDA has chosen NOT to approve Zavesca (miglustat) for the treatment of NPC at this time.  We have learned in our discussions with Actelion representatives that there are three possible outcome scenarios with regard to FDA announcements as it pertains to New Drug Applications:

1)      An Approval Letter

2)      A Non-Approval Letter (straight denial)

3)     Complete Response Letter (which is what Actelion has received)

For more information about the FDA Complete Response Letter ~ please refer to this link:

With the "Complete Response Letter" the FDA 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.  In addition, the CEO of Actelion, Jean-Paul Clozel, MD has indicated that they intend to remain committed to moving forward towards an approval with regards to Zavesca for patients suffering from NP-C.

In 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. 

We will keep you abreast of new developments as we become aware of them ~ WE WILL PERSEVERE!

Kind Regards,


[March 9, 2010 mem]

Newspaper Features FDA Review of Zavesca
Lead Story for Daily Jefferson County Union

The Daily Jefferson County Union ran as the lead story the FDA's review of Zavesca (miglustat) for use in Niemann-Pick Type C patients on February 24.  The Daily Union interviewed NNPDF Board Vice Chair Barb Vorpahl regarding her testimony to the FDA advisory committee and the subsequent review of Zavesca by the FDA. 

The story was triggered by a press release issued by the NNPDF Central Offices, and was then expanded into a full length news feature.  Click here to read the story published by the Daily Union.

Update:  The Daily Union followed up the above story with an editorial highlighting the FDA process, and in support of the years of work and progress made by the NNPDF.  Please click here to read the editorial.

[March 9, 2010 mem]

United States FDA Advisory Committee Backs Use of Zavesca for NPC
Significant Step Toward Possible FDA Approval

Group at FDA Advisory Committee Review
NNPDF Member families, researchers and media attended the FDA advisory committee's review of Zavesca (miglustat) on January 12.
An advisory committee comprised of medical and clinical experts will recommend to the U.S. FDA that Zavesca (miglustat) be approved for use in Niemann-Pick Disease Type C (NPC) patients.  The advisory committee reviewed data and heard statements and testimony from scientists, doctors and NPC families in a daylong review January 12

If the FDA approves the use of Zavesca for NPC, it will be a historic step, as this would be the first authorized treatment for the symptoms of NPC in the United States.

Many NNPDF member families submitted letters to the advisory panel in advance of the hearing, regarding the impact Zavesca has had on their family member, or what the availability of Zavesca might have meant for a loved one already lost to NPC.

Barb Vorpahl, NNPDF Board Chair, addressed the panel on behalf of NNPDF families (read Barb's presentation here), along with Cindy Parseghian of the Ara Parseghian Medical Research Foundation (APMRF) and Phil Marella of Dana's Angels Research Trust (DART).

Dr. Isaac Kobrin, Actelion's chief medical officer, told the panel, "This is a relentlessly progressive disease. We clearly see a change in the progression rate (with Zavesca). It's a very clear signal."

Weighing the risk of the drug versus potential benefits, Katherine Flegal, a senior research scientist at the Centers for Disease Control and Prevention, stated, "The risk from the drug is not devastating, but the disease is devastating. I think patients deserve an opportunity to see if they can benefit."

Reuter's News Service published this article about the hearing and decision.

From the Reuter's article: 

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 FDA usually follows panel recommendations when deciding whether to approve medicines. A decision [from the FDA] is due by March 10.

[Jan 13, 2009 mem]

U.S. Food and Drug Administration Grants Priority Review for Zavesca
Committee Discussion Scheduled for January 12, 2010

A press release issued by Actelion Ltd, makers of Zavesca (miglustat), announced that a supplemental new drug application for an extension of indication for Zavesca  for the treatment of progressive neurological manifestations in NPC has been accepted by the U.S. Food and Drug Administration (FDA).

Further, the FDA has granted Zavesca a priority review designation, given to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.  It also means that the FDA will aim to complete the review within six months.  Read the full press release here.

On January 12, 2010, the FDA's Endocrinologic and Metabolic Drugs Advisory Committee will discuss new drug application (NDA) 21–348, ZAVESCA (miglustat), 100 milligram (mg) capsules, by Actelion Pharmaceuticals, Ltd., proposed for the treatment of progressive neurological manifestations (symptoms related to the nervous system) in patients with Niemann-Pick Disease (type C).  Read more, including how you can submit a written statement or make an oral presentation to the committee, here:

[Dec 1, 2009 mem]

Experimental Treatments for NPC

While there is no definitive therapy for NPC, research is continuing to identify potential treatments that could either slow or stop the progression of the disease.

A clinical trial of Zavesca® (or Miglustat) for NPC has been evaluated and additional consideration of trials is underway in the U.S. and Europe. Zavesca has slowed, but not stopped, neurological decline when tested on NPC mice.

A drug assay was conducted by Dr. Laura Liscum. Nearly 50,000 compounds have been tested by Bristol Meyers Squibb for potential effectiveness with NPC. Fifty compounds were identified as candidates for further testing but none has proven suitable for human use. Work is continuing on related and new compounds in a number of laboratories.

Laboratory studies of neurosteroids have had encouraging results when tested on mice but more work needs to be conducted before a clinical trial can be considered.

Many of the symptoms of Niemann-Pick Type C can be controlled or tempered by drugs and supportive treatment.

Cate Walsh-Vockley, National NP Coordinator, can provide guidance to families in this area. Each patient must be considered individually, depending on symptoms. Supportive treatments should be re-evaluated on a regular basis as the disease progresses.

When Considering Experimental Therapies

Recently, there has been discussion on the listserv and elsewhere about experimental therapies for Niemann-Pick Disease. Some of these therapies are being used in the setting of formal clinical trials, while others are not. In either case, families are faced with considering whether a therapy is right for their affected family member.

Because of this, we have developed a new information sheet for you to use as you think about these issues. It is called "For Your Information - Thinking about Experimental Therapies."

[June 22, 2009 mem]


“The Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the Canadian Chapter of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the CCNNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”