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…
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
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
Apellis Reports Positive Results from Phase 3 Study Comparing Pegcetacoplan (APL-2) to Eculizumab in PNH Patients
January 16, 2020 Apellis Pharmaceuticals, a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, announced positive results from the Phase 3 PEGASUS study evaluating pegcetacoplan (APL-2) in adults with PNH. Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16. The safety profile of pegcetacoplan was comparable with eculizumab in this study. Click here to read the company’s full press release.
January 16, 2020 Akari Therapeutics, a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases, announced positive data from Phase 3 of their CAPSTONE study in complement inhibitor naïve, transfusion-dependent PNH patients. The interim data on the first eight patients, who were all transfusion dependent at entry to the CAPSTONE study, show that all four patients randomized to nomacopan were transfusion independent for the first six months of treatment, while all four patients on standard of care (blood transfusion with or without anticoagulation) remained transfusion dependent. Click here to read the company’s full press release.
Apellis Pharmaceuticals Inc., a biopharmaceutical company focused on the development of novel therapeutic compounds to treat diseases through the inhibition of the complement system, has initiated its PEGASUS Phase 3 clinical trial assessing the safety and efficacy of APL-2, a novel inhibitor of complement factor C3, in patients with PNH compared to eculizumab.
The Guide to Living Well with PNH has been updated to include information on the latest news in PNH research, navigating the complexities of accessing treatment through clinical trials, traveling with PNH, and more! Please click here to read more and to download or order your copy today.
Alexion Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of life-transforming therapies for patients with devastating and rare diseases, recently announced positive results from a phase 3 study of their second generation of Soliris® (ALXN1210), for the treatment of PNH. The press release announcing these results can be read here.
Ra Pharmaceuticals, a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases, recently announced interim results from their ongoing, global Phase 2 study evaluating RA101495 for the treatment of PNH. The press release announcing these results can be read here.
This post is for all patients living in Ontario who might be affected by the new OHIP+: Children and Youth Pharmacare Program. Beginning January 1, 2018, all children and youth 24 years of age or younger in Ontario will be able to get their prescription medications for free by simply showing their Ontario health card number and a prescription. Coverage will be automatic, with no upfront costs.