Frequently Asked Questions – 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.

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.  As of June 15, 2023, Amylyx and the pCPA have successfully concluded negotiations for ALBRIOZA and entered into a Letter of Intent (LOI) for the terms and conditions under which ALBRIOZA would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada.

As of September 1, 2023, following provinces have made decisions regarding the public reimbursement of ALBRIOZA:

• Ontario: With criteria under the Ontario Drug Benefit Formulary (Exceptional Access Program)
• Québec: With criteria under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
• British Columbia: Under BC PharmaCare Formulary (special authorization)
• New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary
• Alberta: Under the Alberta Drug Benefit List
• Nova Scotia: Under the Nova Scotia Pharmacare Program
• Newfoundland and Labrador: Under the Newfoundland and Labrador Prescription Drug Program (NLPDP)
• Manitoba: Under the Manitoba Drug Benefits and Interchangeability Formulary (special authorization)
• Saskatchewan: Under the Saskatchewan Drug Plan Formulary

The remaining provinces have not yet made a reimbursement decision.

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.

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:

• Amylyx Medical Information:
  Medinfo@amylyx.com
  (877) 374-1208 (For the US and Canada Only)
• Amylyx Care Team (ACT) Patient Support Program:
  1-877-710-0711
  support@amylyxcareteam.ca
  amylyxcareteam.ca

Not yet in all provinces. As of June 15, 2023, Amylyx and the pCPA have successfully concluded negotiations for ALBRIOZA and entered into a Letter of Intent (LOI) for the terms and conditions under which ALBRIOZA would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada. It is now up to individual provinces and territories to make a reimbursement decision for the therapy. If negotiations are successful, provincial and territorial drug plans will provide reimbursement for ALBRIOZA.

As of July 25, 2023,the following provinces have made decisions regarding the public reimbursement of ALBRIOZA:

• Ontario: Yes, with criteria under the Ontario Drug Benefit Formulary (Exceptional Access Program)
• Québec: Yes, under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
• British Columbia: Under BC PharmaCare Formulary (special authorization)
• New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary

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.

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:

• Amylyx Medical Information:
  Medinfo@amylyx.com
  (877) 374-1208 (For the US and Canada Only)
• Amylyx Care Team (ACT) Patient Support Program:
  1-877-710-0711
  support@amylyxcareteam.ca
  amylyxcareteam.ca

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

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.

• Amylyx Care Team (ACT) Patient Support Program:
  1-877-710-0711
  support@amylyxcareteam.ca
  amylyxcareteam.ca

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:

• Amylyx Medical Information:
  Medinfo@amylyx.com
  (877) 374-1208 (For the US and Canada Only)
• Amylyx Care Team (ACT) Patient Support Program:
  1-877-710-0711
  support@amylyxcareteam.ca
  amylyxcareteam.ca

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:

• Amylyx Medical Information:
  Medinfo@amylyx.com
  (877) 374-1208 (For the US and Canada Only)
• Amylyx Care Team (ACT) Patient Support Program:
  1-877-710-0711
  support@amylyxcareteam.ca
  amylyxcareteam.ca