COVID-19 & Fabry Disease · 2020. 4. 8. · disorder (LSD) products for the treatment of patients with Hunter syndrome (Mucopolysaccharidosis type II or MPS II), Gaucher disease,

COVID-19 & Fabry Disease

April 2nd, 2020

*This meeting will be recorded

Fabry International Network [email protected] www.fabrynetwork.org


WelcomePresident Fin, Lut De Baere



• Welcome and introduction – President FIN, Lut de Baere

• General information COVID-19 – Prof. Dr. Eyskens

• COVID-19 and Fabry disease – Prof. Germain

• Mental Wellbeing during COVID-19 – Dr. Ali

• Update from our Industry Partners

• Q&A

Agenda



Dr. Nadia Ali, Ph.D Emory University, USA

Expert Panel

Prof. Dominique P. Germain, MD Ph.DNational Center for Fabry disease, France

Prof. dr. Francois EyskensUZA, Belgium



COVID-19Prof. dr. Francois Eyskens, UZA, Belgium

Covid-19 and Fabry disease

Eyskens François, MD, PhD

Metabolic Diseases

CEMA (Center for Inherited Metabolic Diseases Antwerp)

Highly contagious2 days beforesymptoms

Thank you!

Contact:

francois.eyskens@uza/be



COVID-19 & Fabry DiseaseProf. Dominique P. Germain MD PhD National Center for Fabry

disease, France



Mental WellbeingDr Nadia Ali, Ph.D , Emory University, USA



Industry Partners Update

Statement concerning the impact of SARS-CoV-2 (COVID-19) on the supply of treatment for persons with lysosomal storage disorders (LSDs), including Hunter syndrome (Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, Fabry disease and Metachromatic Leukodystrophy (MLD)

At this time, amid the SARS-CoV-2 (COVID-19) outbreak, Takeda continues to foresee no near-term disruption in the supply of Takeda lysosomal storage disorder (LSD) products for the treatment of patients with Hunter syndrome (Mucopolysaccharidosis type II or MPS II), Gaucher disease, Fabry disease and Metachromatic Leukodystrophy (MLD). We are closely monitoring the external environment and are committed to providing updates in a timely manner, as needed.

Takeda’s strategy is to secure product supply continuity for patients through managing adequate levels of inventory and/or alternative suppliers for the production of our medicines. This strategy is generally applied across our global supply chain for key starting materials, excipients, raw materials, APIs, and finished products.

Based on current assessments of our global supply chain, we do not anticipate any potential supply disruption in the near-term due to the SARS-CoV-2 outbreak. Longer-term, it is not yet possible to predict precisely what the impact may be on individual products. We will continue to monitor the situation as it evolves and take all necessary actions in an effort to ensure supply continuity for the people we serve.

Our top priority is to do all we can to make sure our medicines and services continue to reach the patients and physicians who rely on them, while we protect the health of our employees, those who work alongside them, their families and our communities.

For more information on what Takeda is doing in response to the SARS-CoV-2 situation, please visit the Takeda website: https://www.takeda.com/newsroom/featured-topics/coronavirus-efforts/.

Should you have additional questions, please contact the following persons:

• Linda Calandra, Global Corporate Communications, Rare Metabolic Disease, [email protected]

• Fintan Leonard, Global Patient Advocacy Lead, [email protected]

www.takeda.com

Production and supply of medicines

Following the decree by the Italian government on March 22 calling the manufacture of all non-essential products in Italy to stop, Chiesi Group confirmed that production of all Company medicines would continue without interruption at our sites in Italy and abroad and that production would continue at the same high-quality standards. As of today, work is continuing and we are able to deliver medicines under normal production and distribution channels from all of our production plants in Italy, Brazil and France, and our global operations in research and development continue to advance.

Our commitment to supporting the global response

As a Benefit Corporation, Chiesi Group is committed to having a positive impact on society. In addition to the actions that Chiesi Group and its global affiliates have taken thus far to support patients, institutions and healthcare professionals during the pandemic, today the Group has announced a new €3 million fund to support efforts to address this crisis in Italy. This fund will support a range of actions including donations of personal protective equipment, ventilators and sanitizing gel in response to requests from hospitals and other health institutions. The company has also made direct donations of drugs for respiratory diseases to hospitals in China and Italy. Chiesi has also set up a process where team members in Italy can donate "virtual" hours of work to an association of volunteers in Parma to support emergency response. The company converted the donated hours worked into a monetary donation, which was then doubled by the Chiesi Group. In two weeks close to € 70.000 have already been collected.

Clinical Trials

Unigal clinical trials specifically involving Fabry patients, as you now Protalix is the Sponsor and they are working hard together with the study CRO (IQVIA) and all involved vendors to keep the clinical studies and the treatment for the patients as safe as possible based on the restrictions of COVID19. Please, let us know if there are specific aspects related to the management of ongoing studies on which you would like to receive more information and Protalix will be available to provide those info to you.

WeI hope you and all your members are well, even in this challenging period.

www.chiesi.com

COVID-19 FREQUENTLY ASKED QUESTIONS FROM PATIENT & PROFESSIONAL ADVOCACY, INTERNATIONAL - 1ST APRIL 2020

General

Q: What if I am experiencing symptoms of COVID-19 or I believe I was exposed?A: You should contact your healthcare provider and be prepared to provide a list of signs and symptoms so they can guide you appropriately. For additional information, please review your local municipality, county or state public health website, country website such as the Centers for Disease Control (CDC) www.cdc.gov or the World Health Organization (WHO) www.who.int.

Q: I’m worried about an interruption to the supply of my medicineA: Manufacturers of medicines are working to ensure that there are no interruptions to the supply. For any questions regarding access to your medicine, please speak to your healthcare provider.

Q: I am really concerned and would like to speak with someone at Amicus about my issue. How can I do this?A: Please speak with your healthcare provider if you have any questions about your treatment. To contact the Amicus Patient & Professional Advocacy (P&PA) team, please call 1-866-9-AMICUS (866-926-4287) (Toll-Free) or email [email protected] if you are a USresident. For ex-US residents, please email [email protected].

For people living with Fabry disease

Q: Is it safe for me/my loved one to continue going to clinic visits?A: The safety and wellbeing of you and your family is our number one priority. You should work closely with your healthcare providers and the study site team to develop the best disease management plan for you.

Q: I/we need to travel to the study site for clinical trial assessments and treatment. Is it safe to do so?A: This is a rapidly evolving situation with significant potential for travel restrictions and advisories. Amicus is willing to work with the study site and the clinical trial participant to provide alternative transportation when that can be a solution. Depending on your circumstances, yourstudy care team may recommend that you seek care closer to home. Talk to your study site care team about what is best for you.

www.amicusrx.comNP-GA-IN-00010420 April 2020

We hope you, your families, and your communityremain safe and well as the COVID-19 pandemiccontinues to unfold. The health and safety of ourpatient communities and employees is ourhighest priority.

Given the healthcare resources required tomanage COVID-19, a number of clinical trial sitesworldwide have temporarily postponed electiveprocedures, including clinical trial activities.While we remain committed to moving ourclinical programs forward, AVROBIO supports thistemporary reallocation of resources to ensurehospitals can focus on meeting the needs ofpatients with COVID-19. We are monitoring thesituation closely and are working to developcontingencies, such as virtual monitoring, forongoing trial participation. We will also continueto identify interested and potentially eligiblepatients via remote screening so sites canresume in-person screening and enrollment assoon as it is appropriate.

The AVROBIO advocacy team is available forphone calls or videoconferencing at any time. Wewill continue to keep you informed of ourprogress and any changes. Please let us know ifyou have any questions.

www.avrobio.com

During these unusual times it is importantto follow the instructions of your doctorand team, and follow any official guidancethat helps to keep you and your familysafe. Understandably, some clinical trialactivities such as recruiting new patients,are being de-prioritised but the safety ofsubjects already in our trials remains ofparamount importance. Freeline are inclose discussion with our investigators toensure this and are closely monitoring theevolving situation. Freeline remainscommitted to research in Fabry disease.Many activities are continuing where thereis no direct impact of restrictions imposeddue to COVID-19. We look forward toresuming full activities once it isappropriate to do so.

www.freeline.life

www.sangamo.com

www.sanofi.com

www. idorsia.com



• With so many Fabry patients on ACE/ARBS - what is the latest data on how they can influence COVID-19?

• Is it safe for me/my loved one to continue going to hospital visits?

• I’m worried about an interruption to the supply of my medicine?

• What if the centre is not able to guarantee the administration of the therapy?

Q&A



• World Health Organization – WHO

– www.who.int

• Your patient support organisations

– www.fabrynetwork.org/members

• Your national health organizations & authorities

– Europe: https://www.ecdc.europa.eu/en/novel-coronavirus-china/sources-updated

– USA: https://www.hhs.gov/

• Eurordis

– www.eurordis.org/covid19resources

Reliable Sources

http://www.who.int/

http://www.fabrynetwork.org/members

https://www.ecdc.europa.eu/en/novel-coronavirus-china/sources-updated

https://www.hhs.gov/

http://www.eurordis.org/covid19resources



FIN stands in solidarity with everyone affected by the COVID-19 pandemic. Our thoughts are with all of you who

are affected by the virus.

Please do not hesitate to reach out if there is any way that our community can support you and your patients during

this difficult and uncertain time.

Feel free to contact us through email, our website or social media!

Documents

COVID-19 & Fabry Disease · 2020. 4. 8. · disorder (LSD) products for the treatment of patients with Hunter syndrome (Mucopolysaccharidosis type II or MPS II), Gaucher disease,