New Hope for Canadians with Hereditary Angioedema
Canadians living with hereditary angioedema (HAE) may soon have access to a promising new treatment option.
Theratechnologies, a biopharmaceutical company specializing in innovative treatments for rare diseases, has announced plans to seek approval from Health Canada for donidalorsen, an investigational injection therapy designed to reduce the frequency and severity of HAE swelling attacks. The company has secured exclusive rights to commercialize donidalorsen in Canada under a licensing agreement with Ionis Pharmaceuticals, the drug’s original developer.
Donidalorsen Shows Promise in Clinical Trials
Donidalorsen has shown strong results in clinical trials, offering hope for individuals living with this debilitating condition. A Phase 3 trial called OASIS-HAE (NCT05139810) evaluated donidalorsen against a placebo in more than 90 adults and adolescents with HAE. The results demonstrated that donidalorsen was significantly better at reducing swelling attack rates.
Notably, most patients who completed the Phase 3 trial continued receiving donidalorsen as part of an extension study called OASISplus (NCT05392114). Long-term data from this study indicated that patients receiving injections of donidalorsen every one or two months experienced a striking reduction in swelling attacks, with rates dropping by more than 90%.
These outcomes are consistent with findings from earlier clinical trials, including a Phase 2 study (NCT04030598) and its open-label extension (NCT04307381). Together, the data suggest that donidalorsen treatment can lead to durable reductions in swelling attack frequencies for individuals with HAE.
Targeting the Root Cause of Swelling
HAE is characterized by recurring episodes of swelling, which can affect various body parts including the face, limbs, abdomen, and airways. This debilitating condition is caused by excessive production of a signaling molecule called bradykinin.
Donidalorsen works by targeting prekallikrein, a precursor to the enzyme that controls bradykinin production. By reducing prekallikrein levels, donidalorsen aims to normalize bradykinin production and ultimately prevent or significantly reduce the occurrence of HAE swelling attacks.
Expanded Access to Innovative Therapies
The licensing agreement between Theratechnologies and Ionis also grants Theratechnologies the Canadian rights to olezarsen, another experimental therapy developed by Ionis. Olezarsen is under investigation for the treatment of familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG), two rare disorders characterized by elevated levels of fatty molecules in the bloodstream.
“We are pleased to partner with Theratechnologies, bringing together their deep regional expertise in Canada and our shared goal of delivering innovative new medicines to people living with severe and debilitating diseases,” said Brett Monia, PhD, CEO of Ionis.
Theratechnologies is dedicated to acquiring and commercializing innovative therapies for rare diseases in Canada, working collaboratively with international partners to bring these life-changing treatments to patients who need them most.
“The agreement with Ionis is a testament to our team’s capabilities to advance innovation across North America and reaffirms our commitment to be a commercially focused company that delivers sustained top- and bottom-line growth and value for shareholders,” said Paul Lévesque, president and CEO of Theratechnologies.
Donidalorsen continues to undergo review for potential approval in the United States, with a decision expected in August. Applications for approval are also underway in Europe, where Otsuka Pharmaceuticals holds the commercialization rights.
What are the causes of hereditary angioedema (HAE)?
## New Hope for Canadians with Hereditary Angioedema
**Host:** Welcome back to the show! Today, we’re discussing a new potential treatment for hereditary angioedema, or HAE, a rare and debilitating condition that causes recurring episodes of swelling. Joining us is Dr. Alex Reed, a specialist in [Alex Reed Specialization] and a leading expert on HAE. Dr. Alex Reed, thank you for being here.
**Dr. Alex Reed:** It’s a pleasure to be here.
**Host:** Let’s start by explaining what HAE is for our viewers who may not be familiar with it.
**Dr. Alex Reed:** Hereditary angioedema is a genetic condition that leads to excessive production of bradykinin, a signaling molecule that causes blood vessels to leak fluid. This results in painful and sometimes life-threatening swelling attacks that can affect the face, limbs, abdomen, and even the airways.
**Host:** I understand there’s a new treatment on the horizon, donidalorsen. Can you tell us about it?
**Dr. Alex Reed:** Yes, donidalorsen is an investigational injection therapy that has shown promising results in clinical trials. It works by targeting prekallikrein, a protein involved in the production of bradykinin. By inhibiting prekallikrein, donidalorsen aims to reduce the levels of bradykinin and prevent swelling attacks. [[1](https://www.nejm.org/doi/full/10.1056/NEJMoa2402478)]
**Host:** Those trials sound encouraging. Can you share some of the key findings?
**Dr. Alex Reed:** Absolutely. In a Phase 3 clinical trial called OASIS-HAE, donidalorsen demonstrated a significant reduction in swelling attack rates compared to a placebo. Notably, patients who continued receiving donidalorsen in an extension study saw their attack rates drop by more than 90%! [[1](https://www.nejm.org/doi/full/10.1056/NEJMoa2402478)]These results are very encouraging and suggest that donidalorsen could be a game-changer for individuals living with HAE.
**Host:** That’s fantastic news!
**Dr. Alex Reed:** Indeed. Theratechnologies, a Canadian biopharmaceutical company, is now seeking approval from Health Canada to make donidalorsen available to Canadian patients.
**Host:** That’s wonderful news for Canadians who are living with this challenging condition. Dr. Alex Reed, thank you so much for joining us today and providing this important information.
**Dr. Alex Reed:** My pleasure. Let’s hope this promising treatment becomes available soon and brings much-needed relief to Canadians with HAE.
