Rett Syndrome Families Outraged by Canada's Drug Agency Denial of DAYBUE® (trofinetide)

Canadian Rett Syndrome Coalition Demands Justified Reconsideration Under Time-Limited Access Framework

TORONTO, Oct. 21, 2025 /CNW/ - The Canadian Rett Syndrome Coalition - comprising the Rett Syndrome Society of British Columbia (RSSBC), Rett Syndrome Society of Alberta (RSSA), Saskatchewan Rett Syndrome Association (SRSA), Manitoba Rett Syndrome Association (MRSA), Ontario Rett Syndrome Association of Canada (ORSA), and the Association Quebecoise du syndrome de Rett (ASQR) - together with the broader Canadian Rett syndrome community and with the support of the Canadian Organization for Rare Disorders (CORD) expressed deep outrage and disbelief at the Canada Drug Agency's (CDA, formerly CADTH) decision not to reimburse DAYBUE^® (trofinetide) - the first and only approved therapy for Rett syndrome.

Rett syndrome is a devastating, rare genetic neurological disorder that almost exclusively affects girls; even rarer in males, causing the loss of speech, mobility, and independence, and requiring lifelong 24-hour care.^{i, ii} Between 600 and 900 Canadian families live with this condition.ⁱⁱⁱ Health Canada approved DAYBUE in October 2024 under Priority Review, citing statistically significant improvements in communication, motor function, and quality of life.^iv

Yet in August 2025, CDA's final recommendation rejected public coverage, ignoring the regulator's scientific conclusion that DAYBUE offers meaningful clinical benefit - a decision that leaves hundreds of Canadian children and adults with no access to the only available treatment and no hope for improvement.

"This decision is devastating and indefensible," said Durhane Wong-Rieger, President & CEO of CORD. "Prior to the formalized Time-Limited reimbursement pathway, CDA recommended conditional reimbursement or managed access plans for drugs with data uncertainty, such as spinal muscular atrophy (SMA), retinal blindness, and Friedreich's Ataxia. In their review, the Agency accepted that Rett syndrome meets the same criteria but inexplicably refused to apply the same policy applied in similar situations."

Letter and Reconsideration Proposal Submitted

CORD and the Canadian Rett Syndrome Coalition have formally submitted a letter to the CDA requesting an unprecedented, but fully justified reconsideration of the DAYBUE reimbursement recommendation, accompanied by a Time-Limited Managed-Access (TLMA) proposal - a detailed framework showing how other rare disease drugs with uncertain data have received conditional funding.

The TLMA proposal cites precedents where CDA recommended "reimburse with conditions" for rare disorders, including:

• Zolgensma (SMA) – reimbursed with strict eligibility and follow-up rules;
• Brineura (CLN2 Batten disease) – coverage despite single-arm evidence;
• Luxturna (RPE65 retinal dystrophy) – accepted a disease-specific functional scale; and
• Epidiolex (TSC seizures) – approved with caregiver-reported outcomes central to deliberations.

Each case mirrors DAYBUE: short trials, small samples, non-traditional endpoints -- but clinically meaningful improvements in devastating, untreatable diseases.

CDA's Own Findings Justify Conditional Access

In its recommendation, CDA acknowledged that:

• Rett syndrome meets the "significant unmet need" criteria allowing for evidence uncertainty;
• DAYBUE produced statistically significant improvements in core Rett outcomes (RSBQ and CGI-I);
• Side effects were manageable; and
• Post-market data collection is critical to resolve residual uncertainty.

The patient letter urges CDA to convert its negative recommendation into a two-year, time-limited managed-access listing, requiring:

• specialist prescribing and tight eligibility (mirroring the pivotal trial);
• HRQoL and function data at 6-, 12-, and 24-month intervals;
• continuation/stop rules; and
• outcomes-based pricing agreements.

Call to Action

The Canadian Rett Syndrome Coalition is calling on the CDA and all provincial/territorial drug plans to uphold Canada's commitments to rare disease patients and equity in access:

"We will not accept bureaucratic indifference when individuals with Rett syndrome are being denied the only therapy proven to improve their lives," said Wong-Rieger. "A time-limited, managed-access listing is the balanced, evidence-based, and humane solution CDA's own policies permit. We urge CDA to do the right thing - reconsider and reopen access now."

References:

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ⁱ Fu et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020;4:1-14

ⁱⁱ Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8:170216.

ⁱⁱⁱ Acadia Pharmaceuticals Inc. Data on File. Canada prevalence of Rett syndrome. September 2024. 

^iv Health Canada. DAYBUE Product Monograph. October 11, 2024.

SOURCE Canadian Organization for Rare Disorders (CORD) and Canadian Rett Syndrome Coalition

Media Contacts: For Canadian Rett Syndrome Coalition: Sabrina Millson, B.Ed, CCRP, CCRA, President, Ontario Rett Syndrome Association of Canada, Email: [email protected], Phone: [phone], www.rett.ca; For CORD: Durhane Wong-Rieger, PhD, President & CEO, Canadian Organization for Rare Disorders (CORD), Email: [email protected], Phone: [phone], www.raredisorders.ca