PMPRB releases new Guidelines to monitor and review drug prices English Français Français
As per the new Guidelines, the PMPRB will follow a two-step review process. The Initial or Annual Review serves as a screening process. If this step raises concerns, or if a complaint is received from an approved individual or organization, the PMPRB will then start a more in-depth review. During an In-Depth Review the PMPRB staff compare the drug under review to similar drugs and treatments available for the same medical conditions.
The PMPRB plays a unique and important role in the Canadian health system by ensuring that prices of patented medicines are not excessive. The Guidelines provide a transparent and predictable process to be used by PMPRB staff to review drug prices once they enter the market.
Quick Facts
The PMPRB protects consumers by ensuring that the prices of patented medicines in Canada are not excessive and reports on pricing trends.
The PMPRB does not set drug prices in Canada. The PMPRB reviews prices to ensure that Canadian prices are not excessive.
If an in-depth review indicates that the drug could be priced excessively in Canada, PMPRB staff can recommend a hearing to the Chairperson. If a hearing panel determines the price is excessive, they may order a price reduction.
The PMPRB will host seminars/learning sessions to explain the new Guidelines to interested parties and answer questions.
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Vancouver Sun
2 hours ago
- Vancouver Sun
This Canadian skin-care brand is made just for babies
My Petite Coco is a Canadian skin-care brand that's formulated specifically for babies. Founded by Toronto-based physician and formulator Dr. Erica Weste, the brand was created amid her own search as a new mom for safe, effective products suitable for the delicate skin of a baby. We caught up with Dr. Weste to learn more: Q: For those who aren't familiar, what is My Petite Coco? A: My Petite Coco is Canada's only complete baby skin-care collection founded and formulated by a physician mom, with a deep commitment to using premium ingredients. Blending the best of nature and science, our products are intentionally crafted to meet the unique needs of a baby's delicate skin and onwards. Discover the best of B.C.'s recipes, restaurants and wine. By signing up you consent to receive the above newsletter from Postmedia Network Inc. A welcome email is on its way. If you don't see it, please check your junk folder. The next issue of West Coast Table will soon be in your inbox. Please try again Interested in more newsletters? Browse here. We are also proudly made in Canada and certified cruelty-free by Leaping Bunny. We ensure every product reflects the highest standards of gentleness, safety and effectiveness, because babies deserve the best. Q: What makes it unique? A: There are quite a few things that make My Petite Coco unique, but I will share my top three. One, it is founded and formulated by a Canadian physician, me, and inspired by my journey into motherhood. Over many years in clinical practice, I noticed that countless baby skin-care products didn't consider the unique needs of all babies, in such a beautifully diverse country like Canada. While I was expecting my daughter, I decided to create a collection of fragrance-free essentials that would be used from the day she was born. Two, every formula has undergone clinical testing and independent trials to prove that they are hypoallergenic, non-irritating and safe. Clinical studies aren't common in Canadian baby skin care, but we believe in going the extra mile for the little ones. And, three, we use high-quality ingredients like cupuaçu, murumuru, squalane and argan oil, rich in vitamins and essential minerals, to gently hydrate, nourish and protect your baby's delicate skin. Q: Who is the target customer? A: Our target customer is a thoughtful, modern parent — often a mom — who is pregnant or navigating the early months of parenthood. Q: Is there a 'hero' product? A: The Baby Balm is our multitasking hero due to its unique blend of cupuaçu, shea butter and squalane. It works beautifully as a diaper balm, can be used with cloth diapers — no zinc! — and is also great for soothing dry irritated patches of skin from head to toe. Our rich Baby Cream is also quickly becoming a fan-favourite. We've received lots of messages from moms saying that they're using it as a hand cream too, which we love. Q: What is the price range for the products? A: From $18.99 to $25.99 for individual products, and our exclusive full collection offer The Full Bundle retails for $78.99. Q: Where can people learn more? A: Online at Aharris@
Global News
3 hours ago
- Global News
Canada set up a $50M vaccine injury. Those harmed say it's failing them
Kimberly MacDougall lay in a hospital bed beside her injured husband, Stephen, as his final moments came. She and their two kids held him as he took his last breath. Stephen, 45, a service manager for a luxury automobile dealership, had been fighting to live for weeks in May 2021, but stopped struggling. She informed friends on social media that the man she loved, incredibly fit and with no prior health issues, wouldn't make it. Outside the window of his intensive care room in Peterborough, Ont., an impromptu group of friends soon gathered to hold a vigil, in love and support. 'I saw things nobody should see and I wasn't equipped to deal with,' MacDougall remembered four years later. 'I watched them use the paddles on him. I watched them bag him.' A rare adverse reaction to a COVID-19 vaccine left Stephen dead in his prime. Ross Wightman, a former pilot and realtor, social worker Shannon Dupont, and kindergarten assistant Kayla Pollock also suffered life-altering injuries after their vaccinations. These four people, and their families, were among millions of Canadians who rolled up their sleeves to get their shots during the pandemic. For their loved ones, communities and country. The largest public immunization in Canadian history reduced the spread of deadly disease, saving the lives of thousands of Canadians by mitigating the effects of the virus and reducing emergency room admissions. For most, vaccines slowly brought life back to normal. But for a small group injured by their shots, life never returned to what it once was. The 0.011 per cent The government reassured the public that serious side effects were possible, but rare. Advertisement There have been 11,702 reports of serious adverse events following a COVID-19 vaccination, according to Health Canada. That's equal to 0.011 per cent of the 105,015,456 doses administered as of December 2023. As a way to help, then-prime minister Justin Trudeau announced the Vaccine Injury Support Program (VISP) in December 2020. The effort, which began six months later, aimed to support people who have been seriously and permanently injured by any Health Canada-authorized vaccine administered in the country on or after Dec. 8, 2020. Approved claimants could receive lump sum injury or death payouts, ongoing income replacement, and reimbursement of medical expenses. But instead of the government operating VISP, as is done with similar programs in the United States, the United Kingdom, France, and Germany, Canada elected to outsource the work. In March 2021, the government hired Raymond Chabot Grant Thornton Consulting Inc. — now called Oxaro Inc. — to administer the program. The challenges began soon after it launched. A Global News investigation has uncovered complaints that the program has failed to deliver on its promise of 'fair and timely' access to financial support for the injured. This five-month probe is based on more than 30 interviews with injured and ill people, former VISP workers, and attorneys who allege the effort is being mismanaged, leaving claimants feeling angry, abandoned, uncared for, and even abused. 'They promised to take care of us,' MacDougall added. 'They didn't fulfil their promise.' This Global News investigation also revealed: Oxaro Inc., has received $50.6 million in taxpayer money. $33.7 million has been spent on administrative costs, while injured Canadians received only $16.9 million PHAC and Oxaro underestimated the number of injury claims VISP would get, initially predicting 40 per year and then up to 400 valid claims annually. More than 3,000 applications have been filed — of those, 1,700 people are still waiting for their claim to be decided. Some injured applicants say they face a revolving door of unreachable VISP case managers and fundraise online to survive. Some say their applications were unfairly rejected by doctors they've never spoken to or met. Despite decades of calls for a vaccine injury support program, the federal government cobbled it together during a pandemic. View image in full screen As Global News neared publication, a spokesperson for new Liberal Health Minister Marjorie Michel contacted the news organization and provided this statement: 'These allegations are completely unacceptable. The VISP supports people who are vulnerable and need support. I've asked PHAC to find a solution that ensures a responsible use of funds and that people receive the support they need. All options are on the table.' View image in full screen Minister of Health Marjorie Michel rises during Question Period on Parliament Hill in Ottawa, Monday, June 2, 2025. Adrian Wyld / Canadian Press Neither the company nor PHAC were prepared for the surge of claims that arrived, former workers say. Staff were too few and inexperienced to handle them all, other ex-staffers say. One said VISP operations were plagued by many 'bottlenecks.' Others described it as 'chaos.' Oxaro and PHAC declined interview requests. In response to a 15-page list of questions, the company said, 'The VISP is a new and demand-based program with an unknown and fluctuating number of applications and appeals submitted by claimants.' Read the full Oxaro statement HERE. 'The program processes, procedures and staffing were adapted to face the challenges linked to receiving substantially more applications than originally planned,' Oxaro added. 'Oxaro and PHAC have been collaborating closely to evaluate how the program can remain agile to handle the workload on hand while respecting budget constraints.' Advertisement The complexity of the claims filed can also affect processing timelines, Oxaro said. PHAC, meanwhile, said it is reviewing Oxaro's five-year arrangement to administer VISP, which is up for renewal next year. Read the full PHAC statement HERE. The agency also wrote that it is weighing 'concerns raised by claimants and beneficiaries' and factoring in how other countries managed their respective programs. Its goal: learn 'best practices' elsewhere, and ensure the future Canadian program is delivered 'in a fair, efficient and cost-effective manner.' View image in full screen Fair and efficient? Don't talk to Becky Marie Campbell about fair and efficient. Three weeks after her vaccination in April 2021, Campbell, a B.C. school teacher and mother of four, began to feel numbness in her legs while driving down the highway. Soon, she was unable to walk and was subsequently hospitalized. Like several others who became sick after shots, the perfectly healthy and fit Campbell was sent for a psychiatric evaluation when she raised the possibility of a link between her vaccine and illness. View image in full screen British Columbia resident Becky Marie Campbell became gravely ill after her vaccination, but was denied support by VISP even though her own doctor suggested her illness was 'most likely related' to the shot. Images courtesy Becky Campbell A doctor later determined she was of sound mind. As she prepared to leave the hospital after a month-long stay, Campbell said a staff member offered her a second vaccine shot. She cried. She left in a wheelchair, looking emaciated. Campbell then applied to VISP in October 2021. She racked up $20,000 in debt for treatments, medicines, mobility equipment and physiotherapies during her attempted recovery. Campbell's own physician said her shot and illness were 'most likely related.' View image in full screen A partial view of Becky Marie Campbell's Vaccine Injury Support Program application, which was supported by her own physician. Courtesy: Becky Marie Campbell Unidentified VISP physicians, however, rejected her claim on Sept. 6, 2022. They said they found 'no peer-reviewed medical literature' that suggested a 'causal association' between her vaccination and subsequent illness at that time. When she received the news, Campbell burst into tears: 'You didn't call me, you had no appointment with me,' she said, referring to the panel of three unidentified doctors which VISP hired to evaluate her file. 'They weren't part of my case at all, and they decided my fate? That's a little unfair.' 'I believed that living in Canada, I would be taken care of,' Campbell said. 'It wasn't about the money … It was about receiving support from my country. Instead, I felt I was faceless,' Campbell said. She did not appeal. Advertisement View image in full screen Mike Becker of B.C., experienced severe pain, nausea, dizziness and huge blood clots in his right leg after receiving a vaccine in 2021. His right leg is now larger than the left. He is on blood thinners. Images courtesy Mike Becker Mike Becker understands Campbell's anger and resentment. VISP also rejected his application, even though pain and swelling in his leg began immediately following his vaccine shot and got worse every day until he went to the hospital nine days later. His leg sears with burning pain at night from deep vein thrombosis, commonly known as blood clots. The condition caused his right leg to swell like a balloon, four inches larger than the left. He now suffers from dizziness and can no longer work as a carpet and furniture upholstery technician. Becker has had 30 doctor visits since his hospitalization in 2021, and takes blood thinners to avoid death. Becker said VISP denied him support because his hospital hematologist failed to record his blood platelet levels, which would have confirmed causality between his vaccine and clots. The VISP report, which included no physicians' names, acknowledged Becker's illness came shortly after his vaccination, but concluded the shot was 'unlikely' to have caused it. However, VISP added a caveat: should the medical world's understanding of such events evolve and new evidence come to light, 'this case should be revisited and reconsidered.' Unlike Campbell, Becker appealed. In November 2022, he informed his VISP case manager and sent in the necessary appeal paperwork. Nobody then answered his emails for almost two years, he said. Becker would not let it go. Finally, a VISP case manager responded by email. She informed him that his case had been mistakenly closed, according to a copy of the correspondence obtained by Global News. VISP had incorrectly recorded in its computers that there was no appeal, although his case manager knew Becker had appealed and filed the necessary documents. 'I have spoken with my manager and have asked that we fast-track your case so that we can rectify this timelapse of your case,' the new VISP case manager told him in the email. Eleven months later, he says he's still waiting. Becker calls VISP 'a big scam.' 'It's not working to help people injured like me.' Phyisicians saw trouble coming Some experts say things could have turned out differently. For 40 years, physicians and public health officials in Canada had been calling for a federal, no-fault government vaccine injury support program. All other G7 countries, except Canada, had one. View image in full screen Dr. Kumanan Wilson had tried to persuade the federal government to launch a vaccine injury support program in Canada for years but was unsuccessful until the COVID-19 pandemic slammed the country. Trevor Owens / Global News Among those stressing the need for such a program was Dr. Kumanan Wilson, CEO and Chief Scientific Officer of the Bruyère Health Research Institute. His research focuses on immunization and pandemic preparedness. Before COVID-19, Dr. Wilson said he had 'a frustrating set of discussions' with the federal government. Creating a program, he said, 'kept dropping as a priority.' Dr. Wilson said he warned officials about other countries' experiences with the programs. 'You don't want to stand these things up right in the middle of an emergency,' he said, noting it doesn't typically end well. Advertisement Many of VISP's current woes might have been avoided if only it had begun earlier, Dr. Wilson said. Pain and suffering Kimberly MacDougall of Peterborough, Ont., has never spoken publicly about her husband's death until now. Her pain and suffering are easily seen on her face. View image in full screen Kimberly MacDougall lost her husband after he suffered a severe adverse reaction to his COVID-19 vaccine. Patrick Capati / Global News MacDougall's husband, Stephen, then 45, died from myopericarditis post-COVID vaccine, leaving her a young widow of two children, then 9 and 12. She received the maximum death benefit under PHAC guidelines, an amount equal to about three years of Stephen's salary. Still, she believes the sum that PHAC and Oxaro paid out is unjustly low. Stephen expected to work 15 or more years in the luxury car business. He had planned to fund their children's university educations and was a rising star in his world who was being headhunted. View image in full screen A family photo of Kimberly MacDougall, her late husband Stephen MacDougall and their children during happier times. Courtesy of Kimberly MacDougall As a community leader, Stephen had encouraged many people to get vaccinated, but a series of disastrous events followed his immunization: the myopericarditis was a deadly inflammation of both his heart muscle and the lining outside it that claimed him in weeks. MacDougall plunged into grief and trauma. A family friend hired lawyer Lori Stoltz to file MacDougall's VISP claim. View image in full screen The VISP prepared this brochure to explain the process of applying and getting financial support to people injured by COVID-19 vaccines. Global News While VISP brochures advertise that the program will 'continue to support you for as long as needed,' there was a cut-off time for MacDougall and her kids. In addition to the death benefit, the program said it would only pay for enough grief therapy to cover weekly sessions for MacDougall and their children for a little over three months. What's more, MacDougall said, VISP would only pay $100, roughly half the cost of each visit. That's when Stoltz wrote a blistering letter to VISP, saying she was 'stunned' by the program's 'apparent institutional indifference' to the young family's 'suffering and need for financial support.' VISP then conceded it would refund the full cost of each session. But the program dug in — 15 visits only. 'And then my kids are supposed to be fine?' MacDougall said, voice breaking. 'I'm supposed to be fine?' She has been unable to return to work as an elementary school teacher. MacDougall thinks VISP lacks humanity and is 'shameful.' As she mourned her husband's death, she explained, a VISP case manager requested she get copies of his autopsy report and death certificate. 'It got to a point where everything was a battle. I didn't have any fight left in me. And that's kind of where I'm at, that's how I've moved forward,' MacDougall said. 'I don't want to fight anymore.' Advertisement Lengthy delays Toronto attorney Jasmine Daya called for a Vaccine Injury Support Program in November 2020, a month before the official government announcement. Now, she and other attorneys harshly criticize the program. Daya calls it 'a sham.' View image in full screen Lawyer Jasmine Daya wanted the government to create a vaccine injury support program. Now, she thinks what was later created is a frustrating 'sham.' Trevor Owens / Global News She says her numerous emails to VISP often only receive generic responses. 'Sometimes those auto emails say, 'Due to the high volume, we'll get back to you when we can,'' Daya added. 'I want to be able to do my job, which is to help these individuals, and I can't.' Victoria lawyer Umar Sheikh also said VISP is 'incredibly difficult to deal with,' adding its findings are not necessarily reliable or fair to people, and they take too long. A VISP brochure and its staff have told applicants that the average claim can take 12 to 18 months to process. But some have waited far longer. Sheikh is helping several claimants with VISP battles, including Dan Hartman, an Ontario father who lost his 17-year-old son, Sean, in September 2021. The teenager died alone in his bedroom in the middle of the night. View image in full screen The last photo Dan Hartman took with his son Sean before his sudden, unexplained death after his vaccination. Photo courtesy of Dan Hartman Dan Hartman suffered emotional shock, taking time off work. Three physicians from VISP rejected Hartman's first claim in 2022, denying the father's assertion that the vaccine was linked to his son's death. Initially, a post-mortem examination characterized the cause of death as 'unascertained.' The VISP report noted the post-mortem on Sean's body found 'mild R(ight) and L(eft) ventricular enlargement,' which a pathologist described as 'not uncommon in athletic young men.' Sean was a hockey player. But the heart enlargement led Hartman to believe his son had a rare adverse reaction, so he appealed the VISP decision with new evidence in May 2023 and still waits. Fed up, Hartman and Sheikh pressed VISP to explain its lengthy delays. View image in full screen Victoria attorney Umar Sheik is helping several VISP claimants with their battles with the program. Max Trotta / Global News According to an email Global News reviewed, VISP staff replied that they have had trouble finding a forensic pathologist to examine the late Hartman's tissues. Revisiting the case may confirm or disprove any causal link between the teen's vaccination and his death. In the meantime, medical knowledge of adverse reactions has increased. Last month, the U.S. Food and Drug Administration told vaccine makers to expand warning labels on COVID-19 vaccines that would spell out the risks of possible heart injuries that afflict males aged 17-26, like Sean Hartman. Advertisement Health Canada issued a similar warning for 'younger male adults and adolescents' in June 2021. Still, Hartman waits. Kayla's 'nightmare' Kayla Pollock waits, too. After first applying in 2022, Pollock's VISP application remains stuck in 'intake.' Her injuries have not been assessed, she says. She uses a wheelchair because of her transverse myelitis, a condition that involves swelling of her spinal cord and the loss of lower body functions. Medical research has documented hundreds of cases of transverse myelitis following COVID vaccination. As a result of her illness, Kayla is no longer able to work. View image in full screen Kayla Pollock says she suffered a vaccine injury that damaged her spine. She mailed her VISP application in July 2022 and was told it was lost. She resubmitted her claim and said her case remains in 'intake' three years later. Dealing with VISP has been 'hell,' and a 'nightmare,' she says. Trevor Owens / Global News She used to be a kindergarten assistant. Now, she receives Ontario disability support. She lost her townhome. Her son's father is now his primary caregiver, and she sees their boy only on alternating weekends. Pollock said things are so bleak for her that she requires personal support workers and has been offered Medical Assistance in Dying (MAID). In the absence of VISP support, she has been raising money online. 'Unfortunately, it costs me more to be alive than if I were dead,' Pollock says. After waiting for three years, she no longer believes VISP will ever compensate her. Approved... yet still outraged Former pilot and realtor Ross Wightman understands the frustration, anger and desperation of people dealing with the support program. VISP accepted his injury claims, but Wightman remains enraged. He says he often cannot reach anyone at VISP and has had 10 case managers work on his file. Ross Wightman's VISP application was approved. He says he regularly waits months for VISP medical expense refunds. The program is an unfathomable 'dumpster fire,' he says. Courtesy Nicole Wightman Wightman, who lives near Kelowna, B.C., was diagnosed with Guillain-Barré syndrome after his vaccination and applied to VISP in 2021. He received $270,000 in indemnities in 2022, becoming one of the first people to be approved. Guillain-Barré syndrome is a rare neurological disorder that has been linked to COVID-19 vaccinations. The condition causes sudden numbness and muscle weakness when the immune system attacks peripheral nerves. No longer able to work, Wightman awaits a second reassessment of his injuries. His wife left her job to care for him and their two young children full-time. After the one-time injury award, he waited 20 additional months to be approved for a VISP income replacement benefit in 2023. That benefit is capped at $90,000 a year — the maximum for all claimants — though he earned far more as a realtor and former pilot. Advertisement Asked about VISP, he likens it to a 'dumpster fire.' 'I don't know how it could be done worse,' added Wightman. 'There have been times where I have thought about not continuing with some of my therapies just because I'm tired of incurring expenses and eating expenses for such a long time.' Instead, he says the slow pace of financial support forced him, for a time, to turn to the 'bank of family.' Wightman stated at one point that $12,000 in VISP funds were deposited into his bank account, but it took him more than 10 weeks to confirm what that refund was supposed to cover. VISP officials also kept him on tenterhooks for months, waiting for $25,000 in other reimbursements, he added. 'Such poor communication and record-keeping undermine trust in the program and create additional stress for those who depend on it,' added Mounting paperwork, mounting debt As a social worker living in Manitoba, Shannon Dupont thought she knew how to help vulnerable people through a crisis. But she, herself, is now lost and alone, battling VISP. Prior to her vaccine injuries, which occurred after each of her three injections, she had two jobs and made an average of $104,000 a year. View image in full screen Shannon Dupont, of Manitoba, displays all the Vaccine Injury Support Program documents, emails and forms she's gathered. Melissa Ridgen / Global News She says her employer mandated that she be vaccinated. But Dupont suffered a stroke, Bell's palsy, lost half her field of vision in her left eye and dexterity in her hands. She developed an autoimmune disorder that gave her hives. In September 2022, the provincial health authority recommended that Dupont receive no further COVID-19 vaccines. She can no longer work. In her dealings with VISP since 2021, Dupont says she has had nine case managers whose letters, emails and forms cover her entire dining room table. View image in full screen VISP told claimants like Shannon Dupont by email that they would contact them 'quarterly.' Two years after she applied, VISP approved her for a one-time $24,294 injury payout. However, she believes VISP 'missed a significant amount of my injury.' She has since applied for reassessment four times and sought refunds for medical expenses. Many vaccine-injured people have also asked for their cases to be reconsidered. VISP now owes her $180,000, Dupont alleges. As she waited, Dupont says she cashed in her investments and lived on credit cards. In March, she finally started receiving a VISP income replacement benefit of $3,700 monthly, but her battles continue due to confusion between VISP and her health insurer. In December, VISP announced the government program would now be her first payor, reversing its initial position. She says that the insurer now wants her to repay $86,000 — money she says VISP has not paid her.
Cision Canada
3 hours ago
- Cision Canada
OXLUMO® (lumasiran injection) Now Reimbursed in Canada For the Treatment of Primary Hyperoxaluria Type 1 (PH1) in Pediatric and Adult Patients
OXLUMO has been shown to significantly reduce urinary oxalate, which drives the progression of PH1 Disease 1 MISSISSAUGA, ON, July 2, 2025 /CNW/ - Alnylam Canada ULC is pleased to announce that OXLUMO ® (lumasiran) is now funded across Canada through both public and private plans. OXLUMO is an RNA interference (RNAi) therapeutic administered via subcutaneous injection, indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients. 1 OXLUMO is the first therapy for PH1 approved in Canada, and among the first medications to be included on the common list of new drugs for rare diseases, as part of the Government of Canada's National Strategy for Drugs for Rare Diseases. The common list was developed to help patients with rare diseases have access to treatments as early as possible. PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction. 2 Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body. Oxalate overproduction results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis (renal deposition of calcium oxalate crystals), progression to kidney failure, and systemic organ dysfunction. 2 "Funding OXLUMO through the National Strategy for Drugs for Rare Diseases provides a new treatment option for people diagnosed with this debilitating, genetic disease – many of whom are infants and children," said Colleen Coxson, Country General Manager, Alnylam Canada ULC. "I want to congratulate the Canadian government for prioritizing access to therapies for those living with rare conditions, as there are often very limited options for patients." There are several types of primary hyperoxaluria (PH), however, PH1 is the most common and the most severe form, accounting for 70 to 80 per cent of all PH cases. 3 PH1 affects approximately four individuals per million, with some regions – such as the Middle East and North Africa – having a higher genetic prevalence. 4 Symptom onset ranges from early infancy to sixty years of age, with the median age being four to six years. 4 The remainder of affected cases present in adulthood with 20 to 50 per cent presenting late stages of chronic kidney disease when diagnosed. 4 "This funding decision marks a major step forward in the management of hyperoxaluria type 1, offering hope to patients of all ages living with this ultra-rare genetic condition," said Dr. Vladimir Belostotsky, Division Head for Pediatric Nephrology at McMaster Children's Hospital. "The medication has been shown to effectively lower urine oxalate levels, reducing the burden that leads to severe clinical symptoms." The positive recommendation for reimbursement was supported by results of the ILLUMINATE clinical studies, including ILLUMINATE-A: a randomized, double-blind, placebo-controlled clinical study in patients six years and older with PH1, ILLUMINATE-B: a single-arm clinical study in patients less than six years of age with PH1 1 and ILLUMINATE-C: a single-arm trial in patients of all ages with advanced PH1, including patients on dialysis. 5 The ILLUMINATE-A study showed that OXLUMO met its primary endpoint, evidenced by a 53 per cent mean reduction in urinary oxalate, and a 65 per cent mean reduction in urinary oxalate relative to baseline. 1 In ILLUMINATE-B, OXLUMO demonstrated a 72 per cent mean reduction in spot urinary oxalate:creatinine ratio from baseline to month six (averaged from months three to six), meeting its primary endpoint. 1 In ILLUMINATE-C, OXLUMO met its primary endpoint, demonstrating a 33% least squares (LS) mean reduction in plasma oxalate (POx) levels in patients not on dialysis (Cohort A) and a 42% LS mean reduction in POx levels in patients on hemodialysis (Cohort B) from baseline to month six. 6 About OXLUMO ® (lumasiran) 1 OXLUMO ® (lumasiran) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in the liver to deplete the production of the glycolate oxidase (GO) enzyme. By silencing HAO1 and depleting the GO enzyme through RNA interference, OXLUMO in turn reduces the amount of oxalate that is produced. This process helps address the root cause of the rare, genetic disease since an overproduction of urinary and plasma oxalate levels is the underlying cause of PH1. About Primary Hyperoxaluria Type 1 (PH1) PH1 is an ultra-rare genetic disease that is characterized by oxalate overproduction in the liver, causing renal damage. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones. PH1 is associated with a progressive decline in kidney function, which exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and deposition of oxalate in bones, eyes, skin, and heart, leading to severe illness and death. Management options to date have been limited to hyperhydration, crystallization inhibitors and, in a minority of patients with a specific genotype, pyridoxine (vitamin B6). These measures only delay the inevitable progression to kidney failure and the need for intensive dialysis as a bridge to a dual or sequential liver/kidney transplant. Other impacts of the disease include: infants often fail to thrive, meaning they are weak and not growing or developing at a normal rate. 7 Affected children frequently face developmental challenges, with social barriers and the requirement of accommodations to be made at school to meet their special medical needs. 6 About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made.5 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its " Alnylam P 5 x25" strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Canada is headquartered in Mississauga, Ontario with established operations since June 2018.
