COVID-19 & Fabry Disease April 2 nd , 2020 *This meeting will be - - PowerPoint PPT Presentation

COVID-19 & Fabry Disease

April 2nd, 2020

*This meeting will be recorded

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

Welcome President Fin, Lut De Baere

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

• 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

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

• Dr. Nadia Ali, Ph.D

Emory University, USA

Expert Panel

• Prof. Dominique P. Germain, MD Ph.D

National Center for Fabry disease, France

• Prof. dr. Francois Eyskens

UZA, Belgium

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

COVID-19

• Prof. dr. Francois Eyskens, UZA, Belgium

Covid-19 and Fabry disease

Eyskens François, MD, PhD Metabolic Diseases CEMA (Center for Inherited Metabolic Diseases Antwerp)

Highly contagious 2 days before symptoms

Thank you!

Contact: francois.eyskens@uza/be

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

COVID-19 & Fabry Disease

• Prof. Dominique P. Germain MD PhD National Center for Fabry

disease, France

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

Mental Wellbeing

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

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

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

• utbreak. 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, linda.calandra1@takeda.com
• Fintan Leonard, Global Patient Advocacy Lead, fintan.leonard@takeda.com

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 medicine A: 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 PatientAdvocacy@amicusrx.com if you are a US

• resident. For ex-US residents, please email patientadvocacyintl@amicusrx.com.

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, your study 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.com NP-GA-IN-00010420 April 2020

We hope you, your families, and your community remain safe and well as the COVID-19 pandemic continues to unfold. The health and safety of our patient communities and employees is

• ur

highest priority. Given the healthcare resources required to manage COVID-19, a number of clinical trial sites worldwide have temporarily postponed elective procedures, including clinical trial activities. While we remain committed to moving our clinical programs forward, AVROBIO supports this temporary reallocation of resources to ensure hospitals can focus on meeting the needs of patients with COVID-19. We are monitoring the situation closely and are working to develop contingencies, such as virtual monitoring, for

• ngoing trial participation. We will also continue

to identify interested and potentially eligible patients via remote screening so sites can resume in-person screening and enrollment as soon as it is appropriate. The AVROBIO advocacy team is available for phone calls or videoconferencing at any time. We will continue to keep you informed of our progress and any changes. Please let us know if you have any questions. www.avrobio.com During these unusual times it is important to follow the instructions of your doctor and team, and follow any official guidance that helps to keep you and your family

• safe. Understandably, some clinical trial

activities such as recruiting new patients, are being de-prioritised but the safety of subjects already in our trials remains of paramount importance. Freeline are in close discussion with our investigators to ensure this and are closely monitoring the evolving situation. Freeline remains committed to research in Fabry disease. Many activities are continuing where there is no direct impact of restrictions imposed due to COVID-19. We look forward to resuming full activities

• nce

it is appropriate to do so. www.freeline.life www.sangamo.com www.sanofi.com

• www. idorsia.com

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

• 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

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

• 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

Fabry International Network info@fabrynetwork.org www.fabrynetwork.org

COVID-19 & Fabry Disease

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

• ur 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!