Last Update: August 11, 2022

GENERAL

What is ALBRIOZA (AMX0035)?

ALBRIOZA (sodium phenylbutyrate/ursodoxicoltaurine) is indicated for the treatment of people living with amyotrophic lateral sclerosis (ALS).

Sodium phenybutyrate is a histone deacetylase inhibitor that is believed to reduce a mechanism known as endoplasmic reticulum stress (ER stress) that may contribute to motor neuron degeneration in ALS. Ursodoxicoltaurine, also known as TUDCA or TURSO aims to relieve stress on the mitochondria, which produce energy for cells to live. The combination of ER and mitochondrial stress reduction is believed to explain the beneficial effects of ALBRIOZA.

It is available as individual sachets, each containing 10 g of powder (3 g sodium phenylbutyrate and 1 g ursodoxicoltaurine) to be reconstituted in 250 mL of room temperature water and taken orally or administered via feeding tube within one hour of preparation.

The recommended dose in the product monograph is one sachet daily for the first three weeks and one sachet twice daily thereafter.

What is the current status of ALBRIOZA with the drug access pathway?

In June 2022, ALBRIOZA (AMX0035) was approved, with conditions, by Health Canada as a treatment for ALS.

The drug is now commercially available in Canada. Considerations, whether provincial drug plans will cover it, have not yet been determined. As of August 11, 2022, ALBRIOZA is on the list of drugs in active negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA). For more information on the approvals, read the press release from Amylyx.

What is the process for getting a drug approved through Health Canada?

Health Canada conducts a review of the drug to assess its safety, efficacy, and quality. The process begins when the drug manufacturer files a New Drug Submission (NDS) with Health Canada. Health Canada must accept the New Drug Submission before regulatory review begins.

There are three timelines under which Health Canada conducts drug reviews:

  • Standard Review, which is almost a one-year process (300 days)
  • Notice of Compliance with Conditions, which is an approximately 200-day process.
  • Priority Review, which is a six-month process (180 days)

If Health Canada concludes the benefits of the drug outweigh the risks and that the risks can be mitigated, they will issue a Drug Identification Number (DIN) and a Notice of Compliance (NOC).  Health Canada may also issue a Notice of Compliance with conditions (NOC/c). This is authorization to market a drug with the condition that the sponsor undertake additional studies to verify the clinical benefit. An NOC or NOC/c means the drug is approved to market in Canada and physicians can begin prescribing it. At this point, however, the drug is only accessible by paying for it out-of-pocket or through private insurance.

If the drug does not receive a Notice of Compliance, the drug’s manufacturer has the option of providing additional information, re-submitting the application at a later date with additional supporting data, or asking Health Canada to reconsider its decision.

We believe any new innovative ALS therapy should be granted priority review status. Due to the swift progression and terminal nature of the disease, people living with ALS cannot afford to wait almost a year for Health Canada to review a therapy. During this time, people will experience further loss of function, and ultimately, approximately 1,000 Canadians will die of ALS. Visit this blog post on How new drugs become approved and accessible to Canadians for more information.

REIMBURSEMENT AND AVAILABILITY

How can I access the drug now that it has been approved?

ALBRIOZA (AMX0035) is now commercially available in Canada. Once the drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.

If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:

Is the drug available through the public healthcare system/public reimbursement?

Not as of yet. As of August 8, 2022 CADTH has published final recommendations for reimbursement, and the drug is on the list of drugs in active negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA). Following pCPA negotiations, each province/territory does an independent review of the drug and decides if and how the public drug plan will cover the therapy.

ALS Canada will continue to be involved in the CADTH Health Technology Assessment process and is actively working to support the pan-Canadian Pharmaceutical Alliance (pCPA) and the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS. We are hopeful that pan-Canadian and provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for all Canadians who may benefit. There is simply no time to wait for people affected by ALS.

When will the drug be available commercially for purchase?

ALBRIOZA (AMX0035) is now commercially available in Canada. Once a drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.

If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:

Can I get the drug through private insurance?

ALBRIOZA (AMX0035) is currently being reviewed for pricing and reimbursement status. Please speak with your ALS clinician for more information.

Can I pay out-of-pocket for the drug?

Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.

I am not Canadian and I do not live in Canada; can I get ALS treatments shipped to me from Canada?

No. ALS treatments (disease-modifying therapies) are only available through prescription from a Canadian physician.

ALS Canada is aware of the use of online pharmacies, but we do not maintain a list of these pharmacies. There are potential serious risks surrounding the use of online pharmacies. Please visit: Health Canada and US Food and Drug Administration for more information.

Any questions regarding shipment or crossing the border with therapies should be addressed by checking with your country’s customs or food and drug administration authorities for their rules and requirements.

The drug is now commercially available in Canada. Once a drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:

Can I come to Canada for an ALS treatment?

There may be an ALS Society or ALS/MND clinic in the country where you live. Please visit the International Alliance of ALS/MND Associations for a listing of organizations around the world.

Many treatments offered in Canada are also offered in other countries. Please speak with your healthcare provider in your local region to learn about treatment options available to you.

In Canada, there are specialized ALS/MND clinics. A very limited number of these clinics may accept international patients on a referral basis only. It is up to the discretion of the Canadian ALS clinician on whether they prescribe a treatment or not. The ALS Society of Canada cannot comment on wait times and does not provide assistance for medical appointments once in Canada. Costs for all medical appointments including tests (blood work, MRI, etc.), transportation, accommodation, medication, translation services etc. are the responsibility of the individual seeking medical services in Canada.

ALBRIOZA (AMX0035) is now commercially available in Canada. In Canada, please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team Patient Support Program to determine next steps.

Any questions regarding shipment or crossing the border with therapies should be addressed by checking with your country’s customs or food and drug administration authorities for their rules and requirements. If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:

More information:

Travelling for treatment

Each person’s experience with ALS is unique. People living with ALS face different challenges and symptoms of the disease. It is important to be aware of these challenges and to consider both the benefits and risks when deciding on a treatment. This includes travelling abroad. Gathering as much information as possible will enable you to make well-informed decisions for you, and your loved ones.

When making the decision to travel abroad for medical treatments, some key considerations to be aware of both before you go, and when you return include things like, cost, safety, aftercare, rules for travelling with medications, immigration laws and visas.

Please speak with your ALS clinician for more information and consult your government’s healthcare authorities.

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Posted in: Advocacy, Research