News:

REGISTER TODAY: 📢 PNH ADVOCACY WEBINAR – Sept. 10 @ 12:00PM EDT

REGISTER TODAY: Why drug pricing reform in Canada must be stopped to protect PNH patients New federal regulations governing the Patented Medicine Prices Review Board (PMPRB) and the regulation of drug prices in Canada will come into effect on January 1, 2021. On August 4, 2020, the Canadian Association of PNH Patients made a submission in response to the consultation currently underway regarding proposed amendments to the Regulations governing the PMPRB. Despite the most recent changes, as reflected in the June 2020 Draft Guidelines, we remain concerned that the health of Canadians living with PNH continues to be put at risk by the uncertainty generated by the PMPRB reform process to date. On September 10, 2020 at 12:00 p.m. EDT, please join the Canadian Association of PNH Patients and our presenters, Barry Katsof, Founder & President, Canadian Association of PNH Patients and Joanne Koskie, Managing Director, Advocacy Solutions for a free…

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PMPRB Reform Already Having a Detrimental, If Not Deadly, Impact on Patients’ Lives

On August 4, the Canadian Association of PNH Patients made a submission in response to the consultation currently underway regarding proposed amendments to the Regulations governing the Patented Medicine Prices Review Board (PMPRB). Despite the most recent changes, as reflected in the June 2020 Draft Guidelines, we remain concerned that the health of Canadians living with PNH continues to be put at risk by the uncertainty generated by the PMPRB reform process to date.

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Alexion Steps Up to Help Keep PNH Patients Safe

Patients who are part of the OneSource program and rely on public transit can now take a reimbursed ridesharing/taxi service to and from the infusion clinic where they receive their eculizumab treatments. Alexion Canada (the manufacturer of eculizumab) hopes this service will help alleviate any concerns patients may have regarding taking public transit when travelling to and from infusion clinics during this unprecedented time. Details about this program: The OneSource program is now offering travel assistance should a patient indicate that they regularly take public transit to and from their infusion clinic appointments. Patients will choose and book their own car service to and from their appointments. Alexion recommends using Uber, Talixo or Lyft as these companies have implemented strict policies around car cleaning in light of COVID-19. Lyft (Only available to residents in ON and BC) Website: https://www.lyft.com/rider How to use this service: https://help.lyft.com/hc/en-ca/articles/115013079988-How-to-request-a-ride Uber Website: https://www.uber.com/ca/en/ How to…

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New Clinical Trial!

BioCryst Pharmaceuticals is recruiting for a first-in-human study of its novel oral therapy for PNH! BCX9930 is a novel, oral Factor D inhibitor currently in Phase 1 clinical development for the treatment of complement-mediated diseases, including PNH. This three-part study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of BCX9930 with single and multiple ascending doses. BioCryst Pharmaceuticals is currently recruiting both healthy subjects and those with PNH to enroll in the 148-participant study. Clinical trial sites for this study are currently located in South Africa. For more information on this clinical trial, visit ClinicalTrials.gov https://clinicaltrials.gov/ct2/show/NCT04330534… For more information about BioCryst Pharmaceuticals’ Complement Program visit: https://www.biocryst.com/our-program/complement-program/

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COVID-19 for PNH Patients – Update #2

The complete webinar series from Aplastic Anemia & MDS International Foundation regarding COVID-19 is now available to view. While all the webinars contain important information regarding COVID-19, we recommend watching the second webinar as it is focused on patients with PNH and aplastic anemia. Here are the links to the most relevant videos in this series:  Webinar 1Guidance for Bone Marrow Failure Patients to Protect Against Coronavirus (COVID-19)https://www.youtube.com/watch?v=apMyOo7YSeA Webinar 2COVID-19 Update – Focus on Aplastic Anemia and PNHhttps://www.youtube.com/watch?v=I4-KkCyGLGs Webinar 3COVID-19 Update: Pediatric Bone Marrow Failure Patientshttps://www.youtube.com/watch?v=gVgy5-eijC4 Webinar 4COVID-19 Update: Bone Marrow Failure and Infectious Diseaseshttps://www.youtube.com/watch?v=wg4fLuXhyL8 For Aplastic Anemia & MDS International Foundation’s commentary and further information about COVID-19, please visit: https://www.aamds.org/education/covid-19 For regularly updated information on COVID-19 from the Public Health Agency of Canada, please visit: https://www.canada.ca/…/d…/coronavirus-disease-covid-19.html Please follow us on Facebook to stay connected with PNH news.

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COVID-19 for PNH Patients – Update #1

PNH Patients,  Aplastic Anemia & MDS International Foundation has released a webinar for PNH patients regarding COVID-19.  You can watch the free webinar by following this link: https://youtu.be/apMyOo7YSeA For regularly updated information on COVID-19 from the Public Health Agency of Canada, please visit: https://www.canada.ca/…/d…/coronavirus-disease-covid-19.html  

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#FIGHTforourLIVES

Canadian lives depend on access to new medicines. Join the #FightForOurLives movement now and make sure the voice of the PNH community is heard! Visit the CORD website for instructions on how you can get involved in this patient movement. For more information click here You can participate in both official languages – take action and help get the attention of key decision makers around this important issue!  

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Fight for Our Lives!

By now, you will have heard about changes being made to Canada’s Patented Medicine Prices Review Board (PMPRB) and how those changes will impact rare disease patients. New price restrictions will block many rare disease treatments from coming to Canada; whereas governments in other countries have found better ways to lower drug budgets without harming patients. The Canadian Association of PNH Patients joins the rare disease community across Canada in the Fight for Our Lives – urging the federal government to stop the changes to PMPRB and start protecting the lives of patients! Make your voice heard! There is a Twitter campaign happening now to get the attention of key decision makers around this important issue. Send a tweet with the #FIGHTforourLIVES hashtag or use the tweet we’ve provided below. For more information on the Twitter campaign visit the Fight for Our Lives website, click here Click on any of…

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Global Biopharmaceutical Company Alexion Completes Acquisition of Achillion

The acquisition adds two clinical-stage oral small molecule Factor D inhibitors to Alexion’s pipeline and provides the foundation and expertise for a broader oral Factor D inhibition development platform with the potential to treat numerous additional complement-mediated diseases including PNH. Alexion will continue development of Achillion’s oral Factor D inhibitor portfolio, which includes two clinical-stage medicines-in-development – danicopan (ACH-4471) and ACH-5228 – as well as multiple compounds in preclinical development. Phase 3 development is being initiated for danicopan as an add-on therapy for PNH patients with extravascular hemolysis (EVH). Danicopan is also in Phase 2 development for C3G, and ACH-5228 is in Phase 2 development for patients with PNH. Click here to read the company’s full press release  

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Changes to Canada’s Drug Pricing Guidelines Will Hit Rare Disease Patients Hardest

Another article from Canadian health economists concludes that the proposed changes to the Patented Medicine Prices Review Board (PMPRB) guidelines, which come into effect in July 2020, will discourage drug companies from bringing their products to Canada. The revised PMPRB guidelines make no exceptions for therapies to treat rare disorders or other specialized medicines. The worst outcomes are predicted to be significantly reduced access to all new therapies for those patients with either public or private insurance, as well as long delays in accessing new treatment options for rare diseases like PNH. For the full article, please visit: https://www.canadianhealthpolicy.com/products/new-patented-medicine-regulations-in-canada–updated-case-study-.html

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