Catalyst Pharmaceuticals has announced significant international expansion of its rare disease portfolio, with its sub-licensee DyDo Pharma launching FIRDAPSE in Japan and another sub-licensee, Kye Pharmaceuticals, receiving Priority Review designation from Health Canada for AGAMREE.
DyDo Pharma has officially launched FIRDAPSE (amifampridine) Tablets 10 mg in Japan for improving muscle weakness in patients with Lambert-Eaton myasthenic syndrome (LEMS). This launch represents a critical milestone in Catalyst's strategy to expand the global availability of its flagship product.
"We are pleased that our sub-licensee, DyDo, has launched FIRDAPSE in Japan. We believe in health equity, and this launch is a testament to our ongoing efforts to increase patient access to life-changing therapies worldwide," said Richard J. Daly, Catalyst's President and Chief Executive Officer.
FIRDAPSE is the only FDA-approved therapy for LEMS in adults and pediatric patients six years and older. The medication works as a nonspecific, voltage-dependent potassium channel blocker that enhances neuromuscular transmission by increasing acetylcholine release at the synaptic cleft, ultimately improving muscle function in LEMS patients.
LEMS is a rare autoimmune disorder characterized by muscle weakness and fatigue, particularly affecting the proximal muscles of the limbs. The condition significantly impacts patients' mobility and quality of life, making effective treatment options crucial.
Canadian Expansion with AGAMREE
In a parallel development, Catalyst announced that Health Canada has accepted Kye Pharmaceuticals' New Drug Submission (NDS) for AGAMREE, a novel corticosteroid treatment for Duchenne muscular dystrophy (DMD). The submission has been granted Priority Review status, which could expedite the regulatory process with potential marketing authorization before the end of 2025.
If approved, AGAMREE would become the first and only treatment specifically indicated for DMD patients in Canada, addressing a significant unmet medical need. DMD is a genetic disorder characterized by progressive muscle degeneration and weakness, primarily affecting males with symptoms typically appearing in early childhood.
"We are pleased that Health Canada has accepted the New Drug Submission for AGAMREE with a Priority Review, recognizing its potential to address a significant unmet need for patients with Duchenne muscular dystrophy in Canada," stated Richard J. Daly. "In collaboration with our Canadian sub-licensee, Kye, we remain committed to bringing this innovative therapy to DMD patients and ensuring access, particularly for those in underserved communities."
AGAMREE (vamorolone) 40 mg/mL oral suspension received U.S. FDA approval in October 2023 for DMD patients aged two years and older and became commercially available in the U.S. in March 2024. The drug features a unique mode of action based on differential effects on glucocorticoid and mineralocorticoid receptors, potentially offering efficacy comparable to traditional steroids with an improved side effect profile.
Clinical Evidence for AGAMREE
In the pivotal VISION-DMD study, AGAMREE met its primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment. The study demonstrated a favorable safety and tolerability profile, with the most commonly reported adverse events being cushingoid features, psychiatric disorders, vomiting, weight increases, and vitamin D deficiency. These adverse events were generally mild to moderate in severity.
Strategic Global Expansion
Catalyst's international expansion strategy leverages strategic partnerships with regional pharmaceutical companies to bring its rare disease therapies to patients worldwide. Kye Pharmaceuticals already markets FIRDAPSE in Canada for LEMS patients, making it a natural partner for the potential Canadian launch of AGAMREE.
"The launch of FIRDAPSE in Japan marks another advancement in our efforts to expand the geographic footprint of our portfolio products and paves the way for healthcare providers and patients in Japan to access this therapy," noted Daly.
Headquartered in Coral Gables, Florida, Catalyst has been recognized on the 2024 Deloitte Technology Fast 500 List as one of North America's Fastest-Growing Companies and on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies.
About the Sub-Licensees
DyDo Pharma is the rare disease pharmaceutical wholly-owned subsidiary of DyDo Group Holdings, a Japanese company primarily known for its beverage business. DyDo Group Holdings operates through multiple segments, with its Domestic Beverage Business accounting for more than 60% of total sales.
Kye Pharmaceuticals is a growth-stage Canadian specialty pharmaceutical company committed to bringing value to Canadians by identifying, licensing, and commercializing novel prescription medicines. With a growing pipeline of novel medicines, Kye's portfolio spans therapeutic areas including cardiology, psychiatry, pediatrics, rare diseases, hematology, and neurology.
As Catalyst continues to expand its global footprint through these strategic partnerships, patients with rare diseases like LEMS and DMD stand to benefit from increased access to these specialized therapies, fulfilling the company's mission of improving the lives of patients with rare diseases worldwide.
