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Sanofi Genzyme


This page is dedicated to news and information about Sanofi Genzyme’s therapies for use in Niemann-Pick Disease Types A/B and Type B (ASMD). Information is released as it becomes available.

GLOSSARY OF TERMS


About Sanofi Genzyme:

As a pioneer and leader in enzyme replacement therapies, Sanofi Genzyme is committed to finding new therapeutic solutions for patients who may have no other alternatives. Our breakthrough research in genetic engineering and recombinant protein manufacturing has enabled us to develop targeted therapies for several previously untreatable LSDs.  More recently we began clinical trials for an investigational therapy for the treatment of nonneurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick Disease Type B. Though it has taken years of commitment from scientists to begin realizing the potential of gene therapy, we believe it will lead to transformational applications for patients in need of treatment options. For more information , please visit SanofiGenzyme.com..



Phase 1/2 Olipudase Alfa Pediatric Study
Enrollment Completed
.

CLICK HERE FOR COMPLETE UPDATE

04/18/2017 jjb

Sanofi Genzyme Commitment to
Patients with Rare Diseases Highlighted
at WORLDSymposium 2017

.

Support for 31 data presentations helps advance
understanding of lysosomal storage disorders

Sanofi Genzyme, the specialty care global business unit of Sanofi, today announced that new investigational data on its marketed treatments for Fabry disease, Gaucher disease, MPS I and Pompe disease along with data on its rare diseases drug development pipeline will be presented at the 13th Annual WORLDSymposium. For full press release follow the link below.

CLICK HERE FOR FULL PRESS RELEASE

02/09/2017 jjb


Update just in at the NNPDF Central Office from Sanofi Genzyme:

Sanofi Genzyme is investigating olipudase alfa, a novel enzyme replacement therapy for the treatment of nonneurological ASMD. Currently, enrolled patients in the on-going clinical studies are receiving olipudase alfa that is produced in a small scale. Sanofi Genzyme has also developed olipudase alfa which is produced in a larger scale which is intended for commercialization, and which is planned to be used in the clinical trials during the extension phase of the trials. We recently received agency feedback recommending the use of the larger scale commercial olipudase alfa earlier in the clinical studies.

Long Term Study and Phase 1/2 pediatric trial (ASCEND-Peds)

Patients in both clinical trials will continue to receive treatment without any disruptions. Patients in these trials will be switched to the larger scale commercial olipudase alfa once it is available.

Phase 2/3 adult trial (ASCEND)

  1. Patients who are already enrolled in this trial will continue to receive treatment or placebo without any disruptions. The patients who are on treatment will be switched to the larger scale commercial olipudase alfa once it is available.
  1. Newly enrolled patients in the trial, assuming they are randomized to olipudase alfa (and not placebo) will be first dosed with the commercial olipudase alfa on Day 1.
  1. As a result, we have a temporary paused screening of new patients in the ASCEND trial. We anticipate that majority of countries will be able to resume screening efforts in April 2017 with the appropriate country Health Authority approvals for the commercial olipudase alfa.
02/08/2017 jjb

The NNPDF Central Office has just received word that the second site is now open for phase 2/3 clinical trial of Olipudase Alfa for adult patients in the United States.

The second site at University of California San Francisco (UCSF) has been added to clinicaltrials.gov. Dr. Renata Gallagher is the investigator at this site.

CLICK HERE FOR MORE INFORMATION

01/05/2017 jjb

Sanofi Genzyme

First Annual TORCH Awards

We are proud to announce the first annual Sanofi Genzyme TORCH Awards! The TORCH Awards are your opportunity to recognize individuals of all ages who have made a significant contribution to the Lysosomal Storage Disorder (LSD) community. 

Follow the link below for nomination information.

TORCH AWARD NOMINATION INFORMATION

We look forward to recognizing the impact these contributions have made within the LSD community, and thank you for your nominations!

12/15/2016 jjb


Just received in the NNPDF Central Office that the second site has been initiated for phase 2/3 clinical trial of Olipudase Alfa for adult patients in the United States.

The second site is University of California San Francisco (UCSF) and will be added to clinicaltrials.gov soon. Dr. Renata Gallagher is the investigator at this site.

CLICK HERE FOR MORE INFORMATION

12/13/2016 jjb

Dear NNPDF ASMD Patient and Family Community,

The National Niemann-Pick Disease Foundation (NNPDF) is pleased to advise our Niemann-Pick Disease Type A/B & Type B (aka: Acid Sphingomyelinase Deficiency ~ ASMD) Disease patient’s and family community that Sanofi Genzyme has received FDA authorization to begin recruiting for the phase 2/3 clinical trial of Olipudase Alfa for adult patients in the United States (U.S.).   The first U.S. clinical trial site will be at Emory University in Atlanta, Georgia under the direction of Dr. William Wilcox.

Follow the links below to read the full letter along with a press release from July 6, 2016.

We WILL Persevere in our Quest for a Cure!

LETTER TO ASMD COMMUNITY

CLICK HERE FOR FULL REPORT

10/19/2016 jjb

Genzyme

The NNPDF Central Office has received
the following announcment from Sanofi Genzyme regarding the

Acid Sphingomyelinase Deficiency (ASMD)/
Niemann-Pick Disease Type B (NPD B)
Patient-Reported Outcome (PRO) Development and/or Validation
Qualitative Research Study Information

CLICK HERE FOR COMPLETE RESEARCH STUDY INFORMATION

8/11/2016 jjb


The NNPDF Central Office has received
the following press release from Sanofi Genzyme:

Sanofi Genzyme Begins Pivotal Phase 2/3 Trial of Olipudase
Alfa for Adult Patients with Acid Sphingomyelinase Deficiency

CLICK HERE FOR FULL REPORT

07/06/2016 jjb

 

Genzyme

Hello NNPDF Families and Friends,

In July, the NNPDF announced to our Niemann-Pick Type B (ASMD) patient community a new “Qualitative Research Phase” titled:  Patient Reported Outcome (PRO) sponsored by Genzyme.  Patient-Reported-Outcome (PRO) instruments are measures self-reported by patients, about disease symptoms and impact, as well as impact of treatment.

At the 23rd Annual NNPDF Family Support and Medical Conference held in Chicago, Illinois, some individuals of the NPB community were able to take part in the Patient Reported Outcome.  This is what they had to say about their experience:

It was a simple process to do the interview. I was able to open up and discuss anything dealing with NPD. If I didn’t want to discuss it, no problem I could tell them I wasn’t comfortable. If I know that it can help someone else later down the road, it made it all worthwhile.

I was happy to participate, afterwards it gave me a great feeling to be able to share my story and tell just how important it is to get the word out about NPD. It also made me feel that even though we are a small piece of the big picture we still matter and for that we will persevere! 

I feel that the interview was a good thing to have. It let us talk about what it’s like having Niemann- Pick. It was helpful having them ask us questions. It made me think of everything that goes with having it. 

If you have not participated in the “Qualitative Research Phase” titled: Patient Reported Outcome (PRO) sponsored by Genzyme you can still do so!  Please use the contact information below:

US/Canada toll free number:  1-800-257-3157

UK toll free number: 0800-088-5390

Evidera research team at the following
e-mail address:
  ASMDPro@evidera.com

Acid Sphingomyelinase Deficiency
(ASMD)/Niemann-Pick Disease Type B (NPD B)

Patient-Reported Outcome (PRO) Development

Qualitative Research Phase

Patient Interview Study Information

Dateline:  Wednesday, July 22nd, 2015

ABOUT PRO
Patient-Reported-Outcome (PRO) instruments are measures self-reported by patients, about disease symptoms and impact, as well as impact of treatment. When evaluating disease management, quality of care and effectiveness of new treatments, PROs are very important in understanding what is important and meaningful from patients’ perspectives, and how health care interventions can be used to improve patients’ health and health-related quality of life. PROs are increasingly being incorporated into clinical programs, medical practice and in observational research, to evaluate and monitor the impact of medical treatments and health interventions and thus deliver care that is most important and valuable to patients.

AIM OF PRO INITIATIVE
In the light of its commitment to patient-centricity and with the aim to continuously support the patients with Acid Sphingomyelinase Deficiency (ASMD), Genzyme a Sanofi Company has launched an initiative to develop a disease-specific ASMD/ Niemann-Pick Disease Type B (NPD B) PRO instrument. Input from the patient community is a central part of PRO development. In addition to clinical expert interviews and evidence reviews, this research initiative involves also in-depth interviews with patients with ASMD and/or their parents/caregivers. The objective of interviews is to hear patients’ and caregivers’ opinion and perspectives, about the experience with symptoms of ASMD and about the impacts and burden that the disease has on their lives.

VALUE OF NEW PRO MEASURE
The newly developed PRO assessment tool will be available for use widely in various contexts and settings, including clinical research and medical practices. It will provide a comprehensive and standard tool to health care providers to measure severity, progression and overall burden of disease in patients with ASMD, as well as allow assessing and improving quality and effectiveness of care and interventions in a wider sense. This new PRO measure can also be used by researchers and other parties to understand disease impact, severity and progression of disease, as well as the value of treatments and interventions.

PATIENT INTERVIEWS
Patient input is an essential component of this initiative. Interviews will involve a one-hour discussion by an experienced scientist interviewer with patients and/or parents/caregivers.  Participants will be asked questions about disease symptoms, disease impact and general questions about living with ASMD. Interviews will take place via telephone or face-to-face at a location that is convenient for participants. Participation in the interviews is absolutely voluntary and patients and/or parents/caregivers may withdraw their participation at any time.

WHO CAN PARTICIPATE
This research includes adults with ASMD, children older than 7 years of age, as well as parents/caregivers of children with ASMD/NPD B. Your participation in the interviews for PRO measure development study does not prevent you from, or provide preference for, participating in any current or future clinical studies.

CLICK HERE FOR FULL PRESS RELEASE

10/22/15 blg


Genzyme logo

Update from Genzyme on Acid Sphingomyelinase Deficiency (ASMD)
Development Efforts

US Investigational Site Now Open for Recruitment
August 6th, 2015

ALERT: United States NPD Type B Families & Friends

The NNPDF central office noted today that the first investigational site for Genzyme’s Acid Sphingomyelinase Deficiency (ASMD) Pediatric Trial has been updated and is now actively recruiting pediatric patients at the approved clinical trial center in New York, NY.

Families should follow enrollment criteria as provided on the www.clinicaltrials.gov page. To view these criteria, location details and for additional contact information please visit www.clinicaltrials.gov and refer to study reference number: NCT02292654

The title of the pediatric Phase 1/2 trial is listed as: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency.

The ERT pediatric clinical trial site for the United States has been established at Mount Sinai Hospital in New York under the direction of Dr. Melissa Wasserstein and will begin recruiting for pediatric patients diagnosed with ASMD (NPD Type B) within the age ranges listed below:

Patients are encouraged to contact their physicians regarding this information and their physicians will be able to further contact the trial transparency team at Sanofi provided in the www.clinicaltrials.gov clinical trial information page.

CLICK HERE
to read the letter recently sent out to families from the

NNPDF Offices regarding recent Genzyme updates.

 

08/06/15 blg


cap

 

Hello NNPDF Families and Friends,

The NNPDF is pleased to announce to our Niemann-Pick Type B (ASMD) patient community a new “Qualitative Research Phase” titled:  Patient Reported Outcome (PRO) sponsored by Genzyme.  Patient-Reported-Outcome (PRO) instruments are measures self-reported by patients, about disease symptoms and impact, as well as impact of treatment.  Please review the attached announcement which further details the patient interview study and the essential component this information plays in support of the entire ASMD community.

In addition, the NNPDF has been able to work collaboratively with representatives from Genzyme and Evidera (the research consulting firm engaged to oversee this project) will be on-site to conduct “face-to-face” family and patient interviews at the upcoming 23rd Annual NNPDF Family Support and Medical Conference to be held in Chicago, Illinois ~ Thursday, August 6th thru Sunday, August 9th, 2015.

Acid Sphingomyelinase Deficiency (ASMD)/ Niemann-Pick Disease Type B (NPD B) Patient-Reported Outcome (PRO) Development

Qualitative Research Phase

Patient Interview Study Information

Dateline:  Wednesday, July 22nd, 2015

ABOUT PRO

Patient-Reported-Outcome (PRO) instruments are measures self-reported by patients, about disease symptoms and impact, as well as impact of treatment. When evaluating disease management, quality of care and effectiveness of new treatments, PROs are very important in understanding what is important and meaningful from patients’ perspectives, and how health care interventions can be used to improve patients’ health and health-related quality of life. PROs are increasingly being incorporated into clinical programs, medical practice and in observational research, to evaluate and monitor the impact of medical treatments and health interventions and thus deliver care that is most important and valuable to patients.

AIM OF PRO INITIATIVE

In the light of its commitment to patient-centricity and with the aim to continuously support the patients with Acid Sphingomyelinase Deficiency (ASMD), Genzyme a Sanofi Company has launched an initiative to develop a disease-specific ASMD/ Niemann-Pick Disease Type B (NPD B) PRO instrument. Input from the patient community is a central part of PRO development. In addition to clinical expert interviews and evidence reviews, this research initiative involves also in-depth interviews with patients with ASMD and/or their parents/caregivers. The objective of interviews is to hear patients’ and caregivers’ opinion and perspectives, about the experience with symptoms of ASMD and about the impacts and burden that the disease has on their lives.

VALUE OF NEW PRO MEASURE

The newly developed PRO assessment tool will be available for use widely in various contexts and settings, including clinical research and medical practices. It will provide a comprehensive and standard tool to health care providers to measure severity, progression and overall burden of disease in patients with ASMD, as well as allow assessing and improving quality and effectiveness of care and interventions in a wider sense. This new PRO measure can also be used by researchers and other parties to understand disease impact, severity and progression of disease, as well as the value of treatments and interventions.

PATIENT INTERVIEWS

Patient input is an essential component of this initiative. Interviews will involve a one-hour discussion by an experienced scientist interviewer with patients and/or parents/caregivers.  Participants will be asked questions about disease symptoms, disease impact and general questions about living with ASMD. Interviews will take place via telephone or face-to-face at a location that is convenient for participants. Participation in the interviews is absolutely voluntary and patients and/or parents/caregivers may withdraw their participation at any time.

WHO CAN PARTICIPATE

This research includes adults with ASMD, children older than 7 years of age, as well as parents/caregivers of children with ASMD/NPD B. Your participation in the interviews for PRO measure development study does not prevent you from, or provide preference for, participating in any current or future clinical studies.

HOW TO PARTICIPATE

The interviews will be conducted by researchers from a consulting company – Evidera, on behalf of Genzyme. Evidera is required by law to ensure participants’ privacy and to maintain the confidentiality of all patient level data and materials. If you are interested in learning more about this opportunity or participating in these interviews, please reach out to Evidera at the following numbers: US/Canada toll free number:  1-800-257-3157, and UK toll free number: 0800-088-5390. You can also contact the Evidera research team at the following e-mail address:  ASMDPro@evidera.com. You may be enrolled in the study, once your eligibility is established.

The National Niemann-Pick Disease Foundation, Inc. (NNPDF) is not involved in this study and will not have access to any of interview responses.  Representatives from Evidera will be on-site to conduct interviews at the upcoming 23rd Annual NNPDF Family Support and Medical Conference to be held in Chicago, Illinois ~ Thursday, August 6th thru Sunday, August 9th, 2015. 

CLICK HERE FOR FULL PRESS RELEASE

 

[Jul 22nd, 2015 ~ blg]