Galafold posted $521.7 million in sales last year, restricted to patients with specific genetic mutations
Combining Hanmi's long-acting platform with GC Biopharma's enzyme replacement therapy expertise to challenge the legacy intravenous market
U.S. rare-disease specialist BioMarin Pharmaceutical is accelerating its expansion into the Fabry disease treatment market. Just six months after completing its approximately $4.8 billion acquisition of Amicus Therapeutics in April, BioMarin has in-licensed a Fabry disease candidate jointly developed by GC Biopharma (006280) and Hanmi Pharmaceutical (128940).
BioMarin already boasts an oral therapy generating over $500 million in annual sales. Its decision to make an additional bet on an early-stage Korean candidate stems from the therapeutic limitations of existing treatments and the current dominance of intravenous (IV) therapies in the global Fabry market.
An Oral Blockbuster with Limited Patient Reach
Through the Amicus acquisition, BioMarin secured Galafold, an oral Fabry disease treatment. The equity value announced at the time of the deal was approximately $4.8 billion, a transaction that also included Pompe disease treatments Pombiliti and Opfolda.
According to Amicus' financial results, Galafold sales reached $521.7 million, up about 14% year-over-year. It remains the company's flagship product, accounting for roughly 82% of total sales of $634.2 million.
While Galafold offers the convenience of an oral pill, it cannot be used by all Fabry disease patients. Fabry disease is a rare genetic disorder where a specific enzyme is deficient or malfunctioning due to genetic mutations, leading to the accumulation of fatty substances in the kidneys, heart, and nervous system.
Galafold works by stabilizing the activity of the residual enzyme remaining in the patient's body. It can only be prescribed to patients with specific mutations responsive to the drug. Amicus has estimated that roughly 35% to 50% of Fabry patients worldwide carry these responsive mutations.
In an announcement on October 29 (local time), BioMarin CEO Alexander Hardy stated, "This candidate holds the potential to introduce a new treatment paradigm for Fabry disease." He added that by expanding its clinical pipeline through both internal research and external in-licensing, the company expects to reach new patient populations beyond the genetic mutation boundaries currently treatable by Galafold.
Whereas Galafold targets patients with specific mutations via an oral formulation, the newly licensed LA-GLA candidate extends the company's scope to patients requiring Enzyme Replacement Therapy (ERT).
Challenging the €1 Billion IV Market with Monthly Subcutaneous Injections
LA-GLA (HM15421/GC1134A) is a next-generation ERT candidate that directly supplies the deficient enzyme. It combines Hanmi Pharmaceutical's proprietary long-acting platform technology with GC Biopharma's production and clinical expertise in ERT, accumulated through the development and commercialization of Hunterase.
The Fabry ERT market is currently dominated by Sanofi’s Fabrazyme and Takeda’s Replagal. Both products are typically administered via intravenous infusion every two weeks.
Sanofi’s Fabrazyme recorded sales of €1.019 billion in 2025, while Takeda’s Replagal generated 80.4 billion yen in fiscal 2025 (ending March 2026). Although reporting periods and currencies differ, both established treatments command substantial revenues.
LA-GLA differentiates itself through its route and frequency of administration. It is being developed as a subcutaneous (SC) injection administered once every four weeks, replacing IV infusions. If it successfully extends dosing intervals and reduces injection burden—a critical factor for patients requiring lifelong therapy—it could significantly enhance patient convenience while reshaping prescription dynamics in the established ERT market. BioMarin is particularly focused on the candidate's potential to become the world’s first subcutaneous ERT for Fabry disease.
Positioned to capture patients outside Galafold’s scope, LA-GLA also challenges the IV therapy market dominated by Sanofi and Takeda. With the Amicus acquisition, BioMarin secured a commercial revenue base in oral treatments, and this latest agreement adds an ERT candidate with a distinct mechanism of action.
Early-Stage Trial in 18 Patients… $35M Upfront to Each Partner
LA-GLA received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2024 and is currently enrolling patients in a global Phase 1/2 clinical trial.
According to the U.S. clinical trials registry ClinicalTrials.gov, the study commenced on May 14, 2025. It evaluates sequential dosing across low, medium, and high-dose cohorts in 18 Fabry disease patients. Safety, pharmacokinetics, and renal function are monitored for up to 48 weeks, with the trial completion scheduled for August 30, 2028. BioMarin noted that preclinical studies demonstrated differentiated efficacy across biomarkers related to renal function, vascular health, and the peripheral nervous system.
[Source: ClinicalTrials.gov. Status of global Phase 1/2 clinical trials for LA-GLA jointly developed by Hanmi Pharmaceutical and GC Biopharma]
Under the licensing agreement, BioMarin will pay a total upfront fee of $70 million to secure development and commercialization rights. GC Biopharma and Hanmi Pharmaceutical will each receive $35 million. Each company is also eligible to receive up to $202.5 million in milestone payments tied to development, regulatory, and commercial achievements, bringing the potential deal value—excluding royalties—to a combined maximum of $475 million.
Following commercial launch, tiered royalties on net sales will also be paid. BioMarin will assume responsibility for subsequent global development (excluding South Korea), manufacturing, and commercialization following the ongoing Phase 1/2 trial, while GC Biopharma and Hanmi Pharmaceutical retain domestic regulatory and commercial rights.
With BioMarin taking the helm for downstream development and global commercialization, LA-GLA enters its next phase toward entering the global Fabry disease market. Subsequent clinical data will determine how effectively the jointly developed technology of Hanmi and GC Biopharma can challenge existing intravenous treatments.
