New Drug Submission for AGAMREE® (vamorolone) Accepted for Priority Review by Health Canada for the Treatment of Duchenne Muscular Dystrophy

Rhea-AI Summary

Kye Pharmaceuticals has announced that Health Canada has accepted their New Drug Submission (NDS) for AGAMREE® (vamorolone) with priority review status for treating Duchenne muscular dystrophy (DMD). If approved, AGAMREE® would become the first and only Health Canada-approved therapy for DMD, with potential marketing authorization by the end of 2025.

The submission follows AGAMREE's approvals in the US (2023) and European Union (early 2024). According to the Canadian Neuromuscular Disease registry, over 800 boys and young men in Canada live with DMD, a rare disease causing muscle weakness due to lack of dystrophin protein.

The drug's development included clinical trials across five Canadian sites, contributing to its global approval. Patient organizations, including Defeat Duchenne Canada and Muscular Dystrophy Canada, have welcomed this development, highlighting the current lack of approved treatments in Canada.

Positive

• First potential Health Canada-approved treatment for DMD
• Priority review status granted, accelerating approval timeline
• Already approved in major markets (US and EU)
• Addresses an unmet medical need for 800+ Canadian patients

Negative

• Marketing authorization not guaranteed
• Timeline to potential commercialization extends to end of 2025

Insights

Pharmaceutical Regulatory Expert

Health Canada's priority review acceptance for AGAMREE® (vamorolone) represents a significant regulatory milestone for Catalyst Pharmaceuticals, extending their global reach through partnership with Kye Pharmaceuticals. This follows established regulatory success with prior approvals in the US (2023) and EU (early 2024), significantly de-risking the Canadian review process.

The priority review designation is particularly noteworthy, as Health Canada reserves this accelerated pathway for treatments addressing serious conditions with therapeutic options. This designation typically shortens the review timeline and signals regulatory recognition of the drug's potential importance, increasing approval probability before year-end 2025.

If approved, AGAMREE® would secure the coveted first-to-market position in Canada for DMD-indicated therapies, establishing important market positioning. While the article mentions a partnership between Catalyst and Kye, it doesn't detail the commercial arrangement, leaving questions about revenue distribution and commercialization responsibilities.

The positive reception from multiple stakeholders—including patient advocacy organizations and key opinion leaders like Dr. Jean Mah—creates a favorable environment for regulatory approval. The explicit mention of Canadian clinical trial participation further strengthens the submission, as Health Canada typically values local patient data in their review process.

Rare Disease Market Analyst

This regulatory progress represents a modest but positive commercial development for Catalyst Pharmaceuticals. The Canadian DMD market, with approximately 800 patients, offers a controlled expansion opportunity following established commercialization in larger US and EU markets.

For rare disease therapeutics like AGAMREE®, even patient populations can generate meaningful revenue due to premium pricing structures typical in orphan drug markets. The first-mover advantage in Canada is particularly valuable, as it allows Catalyst to establish relationships with the concentrated physician network treating DMD before potential competitors enter.

The statements from multiple patient advocacy organizations (Defeat Duchenne Canada and Muscular Dystrophy Canada) indicate strong support from key stakeholders who often influence both regulatory decisions and subsequent reimbursement pathways. This community endorsement typically accelerates adoption following approval.

The article highlights AGAMREE®'s differentiated profile from standard corticosteroids, addressing side effect concerns that have been challenging for DMD patients. This therapeutic advantage should support favorable formulary positioning and potentially premium pricing when negotiating with Canadian provincial health authorities.

While this development won't dramatically alter Catalyst's near-term financial profile given the market size and timeline (potential approval by end-2025), it demonstrates effective execution of their global expansion strategy and reinforces their position in the DMD treatment landscape.

• Duchenne muscular dystrophy is a rare, debilitating and life-shortening neuromuscular disease¹
  
  
• Upon approval, vamorolone would be the first and only treatment option indicated for patients diagnosed with Duchenne muscular dystrophy in Canada
  
  
• The Canadian regulatory submission of vamorolone follows approvals in the US in 2023, and the European Union in early 2024
  
  
• This New Drug Submission reflects Kye Pharmaceuticals' commitment to advancing treatments for rare diseases and serving the unmet needs of Canadian patients

MISSISSAUGA, ON, April 8, 2025 /PRNewswire/ - Kye Pharmaceuticals, Inc. ("Kye") announced today it has submitted a New Drug Submission (NDS) to Health Canada for the regulatory review and approval of AGAMREE^® (vamorolone). If approved, AGAMREE^® would be the first and only therapy approved by Health Canada with an indication for the treatment of Duchenne muscular dystrophy (DMD). Health Canada has granted AGAMREE^® a priority review and marketing authorization could be granted before the end of 2025. News of the AGAMREE^® priority review was welcomed by national patient organizations.

"Defeat Duchenne Canada is thrilled that Health Canada has accepted AGAMREE^® for priority review. There are currently no approved treatments available in Canada for young people living with Duchenne muscular dystrophy. We urge Health Canada to approve AGAMREE^® to ensure all Canadians for whom this treatment would be beneficial, can access it as soon as possible." Lisa McCoy, Chief Executive Officer, Defeat Duchenne.

"Muscular Dystrophy Canada (MDC) is pleased that Health Canada has granted Priority Review for AGAMREE^® (vamorolone).  While corticosteroids have been a key treatment for decades, they are not currently approved for DMD, and their significant side effects have been a challenge for the Duchenne muscular dystrophy community. If approved, this promising treatment will provide options for the Duchenne muscular dystrophy community." Stacey Lintern, Chief Executive Officer, MDC.

The Canadian Neuromuscular Disease registry estimates that there are more than 800 boys and young men living with DMD in Canada and coping with its debilitating symptoms.² Duchenne muscular dystrophy (DMD) is a type of muscular dystrophy that causes weakness and a wasting of the muscle due to the absence of a protein called dystrophin. If the body is lacking dystrophin, muscle cells become damaged and movements that are considered natural such as getting up from the floor and walking become challenging. Boys with DMD lose the ability to walk in early teens and have cardiac and respiratory difficulties. Bone thinning and scoliosis are also common for those with Duchenne.¹

"Health Canada granting vamorolone a priority review is a promising first step for patients with Duchenne having access to approved medicines in Canada," said Dr. Jean Mah, Director of the Pediatric Neuromuscular Program at the Alberta Children's Hospital in Calgary. "If approved, vamorolone will provide Duchenne families access to an approved therapy that will positively impact their disease. We would like to thank the patients and families from DMD communities across Canada who have contributed to advancing vamorolone research."

This initial step in the review process is a culmination of years of research, including clinical trials of vamorolone conducted in Canada.³ Kye commends the courage of patients and families who participated in these studies, including five clinical trial sites in Canada, generating the data to support the approval of this novel treatment in countries around the world.

"This submission is an important milestone for DMD treatments in Canada and we are grateful for our close partnership with Catalyst Pharmaceuticals," stated John McKendry, President and CEO at Kye Pharmaceuticals. "We look forward to working collaboratively with the DMD community and Health Canada throughout the priority review process."

About AGAMREE^® (vamorolone)

AGAMREE's unique mode of action is based on differential effects on glucocorticoid and mineralocorticoid receptors and modifying further downstream activity. As such, it is considered a novel corticosteroid with dissociative properties in maintaining efficacy that has the potential to demonstrate comparable efficacy to steroids, with the potential for a better-tolerated side effect profile. This mechanism of action may allow AGAMREE^® to emerge as an effective alternative to the current standard of care corticosteroids in children, adolescents, and adult patients with DMD. In the pivotal VISION-DMD study, AGAMREE^® met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.⁴

About Kye Pharmaceuticals

Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines that may not otherwise be available to patients across Canada. With a growing pipeline of novel medicines, Kye's portfolio spans a range of therapeutic areas, including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology. Kye Pharmaceuticals is a private company headquartered in Toronto focused on bringing medications to the Canadian market which fulfill clinically significant unmet needs. Kye is committed to licensing and launching medicines that matter by delivering better outcomes to our partners, Canadian healthcare professionals, and, most importantly, patients across Canada.

For more information about the company, its management, portfolio and pipeline, please visit  www.kyepharma.com

About Catalyst Pharmaceuticals

Catalyst Pharmaceuticals, Inc. (Nasdaq: CPRX) is a biopharmaceutical company committed to improving the lives of patients with rare diseases. With a proven track record of bringing life-changing treatments to the market, Catalyst is focused on in-licensing, commercializing, and developing innovative therapies. Guided by its deep commitment to patient care, Catalyst prioritizes accessibility, ensuring patients receive the care they need through a comprehensive suite of support services designed to provide seamless access and ongoing assistance. Catalyst maintains a well-established U.S. presence while actively seeking to expand its global commercial footprint through strategic partnerships. Catalyst, headquartered in Coral Gables, Fla., was recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's fastest-growing companies.

For more information, please visit Catalyst's website at www.catalystpharma.com.

References:

1. National Institute of Neurological Disorders and Stroke. Muscular dystrophy: hope through research. Available at https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Hope-Through-Research/Muscular-Dystrophy-Hope-Through-Research [last accessed March 2021]
2. Hodgkinson et al. The Canadian Neuromuscular Disease Registry 2010–2019, Journal of Neuromuscular Diseases 8 (2021) 53–6
3. Worsfold, N. (2020, June 10). Defeatduchenne.ca. Available at Defeat Duchenne Canada: $1M Grant Towards Clinical Trial – Defeat Duchenne Canada
4. Dang UJ et al. (2024) Neurology 024;102:e208112.
   doi.org/10.1212/WNL.0000000000208112

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SOURCE Kye Pharmaceuticals Inc.

FAQ

When could AGAMREE (vamorolone) receive approval from Health Canada for DMD treatment?

Marketing authorization for AGAMREE could be granted before the end of 2025, according to the priority review timeline.

How many patients could benefit from AGAMREE in Canada?

Over 800 boys and young men living with Duchenne muscular dystrophy in Canada could potentially benefit from this treatment.

Where else has AGAMREE already received regulatory approval?

AGAMREE has been approved in the United States in 2023 and in the European Union in early 2024.

How many clinical trial sites in Canada participated in AGAMREE's development?

Five clinical trial sites in Canada participated in the development of AGAMREE.