Xenpozyme
There are no current clinical trials for ASMD in the U.S. at this time. For information on Xenpozyme, approved for the treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients, visit XENPOZYME® (olipudase alfa-rpcp)
Latest News
U.S. FDA approves Xenpozyme™ (olipudase alfa) for treatment of ASMD
We are pleased to share the U.S. Food and Drug Administration (FDA) has approved Xenpozyme™ (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients! Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease.
This has been a long road and the ASMD community played a vital and persistent role in bringing this medicine through the trials process and to an approval. We are incredibly pleased that we now have a first approved medicine in the Niemann-Pick community.
Read Sanofi press release.
Read FDA press release.
Read Xenpozyme press release.
08/31/2022
European Approval! News from Sanofi on Xenpozyme (olipudase alfa)
We are thrilled to share this exciting news! Sanofi released the following news today: “Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD”. The European Commission (EC) has approved Xenpozyme® (olipudase alfa) as the first and only enzyme replacement therapy for the treatment of non-Central Nervous System (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with ASMD type A/B or ASMD type B. We will continue to update you as information becomes available.
06/28/2022
News from Sanofi on Xenpozyme (olipudase alfa):
Sanofi released the following news today: “CHMP recommends approval of Xenpozyme® (olipudase alfa), the first and only treatment for ASMD”. This includes the news that the European Medicines Agency (EMA) has adopted a positive opinion for Xenpozyme to be approved in the European Union for the treatment of non-central nervous system manifestations of ASMD in pediatric and adult patients. The press release also shares that the FDA has extended its review in the US by 3 months with a new date for an FDA decision of Oct 3, 2022. We will continue to update you as information becomes available.
05/20/2022
Xenpozyme® (olipudase alfa) approved in Japan, first and only approved therapy indicated to treat acid sphingomyelinase deficiency
03/28/2022
Sanofi Update
Sanofi has shared an important update on olipudase alfa: Olipudase alfa shown to provide sustained improvement across multiple clinical manifestations of ASMD.
02/09/2022
Sanofi Genzyme Update
This morning Sanofi shared their Q1 Earnings Report which includes an important update on the Olipudase Alfa program:
The FDA accepted for Priority Review the Biologics License Application (BLA) for olipudase alfa for the proposed indication as an enzyme replacement therapy for long-term treatment of non–central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in pediatric and adult patients. The target action date (PDUFA) for the FDA decision is July 3, 2022. Historically known as Niemann-Pick disease (NPD) type A and type B, ASMD is an ultra-rare disorder that affects both children and adults. The estimated prevalence of ASMD is approximately 2,000 patients in the U.S., Europe (EU5 countries) and Japan. Due to the rarity of the disease, many patients go undiagnosed or experience delays before receiving an accurate diagnosis, often while the health complications of ASMD continue to progress. Olipudase alfa has received special designations from regulatory agencies worldwide, recognizing the innovation potential of this investigational therapy. Regulatory submissions for olipudase alfa are currently under review in Japan and the European Union. If approved, it will become the first and only therapy available for the treatment of ASMD.
02/04/2022
Sanofi Genzyme Update
Sanofi Genzyme has shared an update about the managed access program (MAP) cohort for olipudase alfa in the United States for certain patients with ASMD via ClinicalTrials.gov. Click here for complete announcement.
05/16/2021
Sanofi Genzyme Update on Olipudase alfa
Sanofi Genzyme has shared an update on compassionate use program for olipudase alfa. Click here for complete announcement.
04/07/2021
Sanofi Issues Press Release on Olipudase alfa
Sanofi Genzyme has issued the following release: Olipudase alfa demonstrated significant improvement in lung function and spleen volume in patients with ASMD. Click here for complete announcement.
10/27/2020
Sanofi Response to ASMD Community in the US
In response to NNPDF’s inquiries to Sanofi about access to olipudase alfa in the US, Sanofi has written the following response.
Click here to read the response.
07/22/2020
Sanofi Genzyme: COVID-19 Study FAQ
In response to questions we have received from you about challenges faced in trial participation during COVID19, Sanofi Genzyme has provided the following information. Click here for complete information.
06/11/2020
Sanofi announces positive topline results demonstrated by olipudase alfa, first and only investigational therapy in late-stage development for ASMD
Olipudase alfa, an investigational recombinant human acid sphingomyelinase, demonstrated positive results in two separate clinical trials evaluating olipudase alfa for the treatment of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.
Click here for complete announcement.
01/30/2020
NNPDF Industry Update Webinar Series featuring Sanofi Genzyme
Click herefor recording.
04/04/2019
ASCEND and ASCEND-Peds Studies Complete Enrollment
Sanofi Genzyme would like to announce that their registration study (ASCEND Study) for adult patients with ASMD Types B and A/B and their pediatric study (ASCEND-Peds) also treating Type B and A/B ASMD pediatric patients have successfully completed enrollment for the targeted number of patients for each of these trials.
Click here for complete announcement.
12/21/2018
Shining a Light on Diagnosis for Rare Genetic Disease Patients
The Lantern Project from Sanofi Genzyme and PerkinElmer expands access to free diagnostic genetic testing for a number of lysosomal storage disorders where tens of thousands go undiagnosed.
CLICK HERE FOR COMPLETE ANNOUNCEMENT
10/23/2018
Update on ASCEND Clinical Trial Sites
We are pleased to inform you that the Phase 1/2 trial in pediatric patients (ASCEND-Peds) is ongoing and still recruiting. Olipudase alfa, an investigational compound, is being developed for the treatment of adult and pediatric patients with nonneurological manifestations of ASMD.
ASCEND-Peds is a multi-national, multi-center, open label, ascending dose trial to evaluate the safety, tolerability and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 64 weeks in pediatric patients with ASMD, specifically Niemann-Pick disease Type B. The primary objective of the Phase 1/2 study is to assess the safety and efficacy of olipudase alfa. For more information please visit clinicaltrials.gov, NCT02292654 or clinicaltrialsregister.eu; EudraCT Number: 2014-003198-40
Sanofi Genzyme is currently conducting three clinical studies of olipudase alfa– a Phase 1/2 study in pediatric patients, a Phase 2/3 study in adult patients and a Long Term study. There are currently no approved treatment options for patients with ASMD.
11/20/2017
Update on ASCEND Clinical Trial Sites
We are pleased to inform you that all the four US clinical trial sites for the Phase 2/3 pivotal study (ASCEND) have been initiated and are currently recruiting patients.
ASCEND is a multi-national, multi-center, double-blinded, placebo-controlled trial to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in adult patients with ASMD, specifically NPD B . The primary objective of the Phase 2/3 study is to assess the safety and efficacy of olipudase alfa. For more information please visit https://clinicaltrials.gov, NCT02004691.
Sanofi Genzyme is currently conducting three clinical studies of olipudase alfa– a Phase 1/2 study in pediatric patients, a Phase 2/3 study in adult patients and a Long Term study. The Phase 1/2 study in pediatric patients is on-going. There are currently no approved treatment options for patients with ASMD.
10/09/2017
ASCEND Trial Update
Sanofi Genzyme is pleased to inform you that the pivotal Phase 2/3 pivotal study (ASCEND), evaluating the investigational therapy olipudase alfa in adult patients with acid sphingomyelinase deficiency (ASMD), has restarted the screening process. ASCEND is a Phase 2/3 multi-national, multi-center, double-blinded, placebo-controlled trial to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of olipudase alfa administered intravenously once every 2 weeks for 52 weeks in adult patients with ASMD, specifically NPB. The primary objective of the Phase 2/3 study is to assess the safety and efficacy of olipudase alfa. For more information please visit https://clinicaltrials.gov, NCT NCT02004691.
Sanofi Genzyme is currently conducting three clinical studies of olipudase alfa – a Phase 1/2 study in pediatric patients (https://clinicaltrials.gov, NCT02292654), a Phase 2/3 study in adult patients and a Long Term study (https://clinicaltrials.gov, NCT NCT02004704). The Phase 1/2 study in pediatric patients have completed enrollment. The Long Term study enrollment is on-going.
06/17/2017
ASCEND Trial Resumes in Atlanta and San Francisco Third Site in Minneapolis Now Open for Enrollment
Just a reminder that recruitment for the ASCEND trial for efficacy and safety of enzyme replacement therapy in adults with ASMD/Type B Niemann-Pick disease has resumed enrollment in Atlanta and San Francisco. A third site in Minneapolis is now open for enrollment as well. Please see clinicaltrials.gov using NCT02004691 as the trial identifier for more information. Please contact the NNPDF office with any questions about enrolling in the trial.
06/28/2017
Phase 1/2 Olipudase Alfa Pediatric Study Enrollment Completed
CLICK HERE FOR COMPLETE UPDATE
04/18/2017
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
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)
- 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.
- 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.
- 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
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 toclinicaltrials.gov. Dr. Renata Gallagher is the investigator at this site.
CLICK HERE FOR MORE INFORMATION
01/05/2017
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
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
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!
10/19/2016
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
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
07/06/2016
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
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:
- Birth to under 6 years of age
- 6 years of age to under 12 years of age
- 12 years of age to under 18 years of age
- More information is detailed at the trial page link here.
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
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.
[Jul 22nd, 2015 ~ blg]