Novo Nordisk's hemophilia A treatment 'denecimig' wins ODD

'Item Ga' list now expanded to include eight items…Novartis' ianalumab newly listed to the 'Item Na' orphan drug list

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Novo Nordisk's hemophilia A treatment 'denecimig (injection)' and Novartis's 'ianalumab (injection)' for moderate-to-severe adult Sjögren's disease have been designated as orphan drugs.

On the 1st, the Ministry of Food and Drug Safety (MFDS) officially announced these new designations. Through this revision, both ingredients have been officially listed on the orphan drug list under Item Ga (Table 1) and Item Na (Table 2) of Article 2, Subparagraph 18 of the Pharmaceutical Affairs Act, respectively, providing novel therapeutic alternatives in the rare disease market in South Korea.

First, 'denecimig (injection)', added as the 8th ingredient under Item Ga (Table 1) of the Pharmaceutical Affairs Act, is a novel candidate being developed by Novo Nordisk to address hemophilia A, a prominent rare hemorrhagic disorder. Under current domestic regulations, 'Item Ga' classifications are reserved for therapies targeting rare conditions officially designated by the Ministry of Health and Welfare (MOHW) under the 'Rare Disease Management Act'. The regulatory inclusion of denecimig is expected to significantly broaden clinical choices for domestic hemophilia A patients who face a lifelong risk of spontaneous hemorrhage and subsequent articular complications.

The inclusion of denecimig expands Item Ga (Table 1) orphan drug list of the MFDS comprising eight active ingredients. The current Item Ga list already includes 'nipocalimab (injection)' for generalized myasthenia gravis, which causes systemic muscle weakness; 'pegunigalsidase alfa (injection)' for Fabry disease, a disorder where intracellular glycolipid accumulation leads to renal and cardiac impairment; and 'onasemnogene abeparvovec (injection)' for infantile, pediatric, bulbar, and adult-onset spinal muscular atrophy (SMA), which induces muscle wasting via motor neuron degeneration.

The list has been expanded to include eight items, including 'garadacimab (injection)' for C1-esterase inhibitor deficiency, which triggers acute subcutaneous and mucosal edema; 'miglustat (capsule)' and 'cipaglucosidase alfa (injection)' administered in combination for Pompe disease, which causes progressive muscle weakness; 'vamorolone (oral suspension)' for severe muscular dystrophy, a pediatric genetic disorder characterized by progressive muscle loss; and 'acoramidis hydrochloride (tablet)' for transthyretin amyloid cardiomyopathy (ATTR-CM), where abnormal amyloid fibril deposition leads to heart failure, alongside the newly added hemophilia A treatment denecimig (injection).

Additionally, 'ianalumab (injection)', listed under No. 409 in Item Na (Table 2) of the Pharmaceutical Affairs Act, was developed by Novartis as a treatment for patients with moderate-to-severe adult Sjögren's disease. The "Item Na" designation is granted to medicinal products targeting diseases with a domestic prevalence of 20,000 or fewer individuals, where no appropriate alternative treatment is available or where the candidate demonstrates a significantly improved safety or efficacy profile compared to existing standards of care.

Sjögren's disease is a systemic autoimmune disorder characterized by chronic exocrine gland inflammation accompanied by severe dry eyes, dry mouth, and arthralgia. Yet, there has been a significant unmet medical need due to the absence of targeted, disease-modifying therapies. As a monoclonal antibody injection, ianalumab targets and inhibits B cells driving the autoimmune response, anticipated to mark a significant turning point for patients who previously lacked viable therapeutic options.

An MFDS official said, "This revision is a measure to provide safe and effective therapeutic options for patients with hemophilia A and Sjögren's disease, who have faced hardships due to limited local patient populations or a lack of alternative treatments. The official added that the ministry will continue to actively use the Item Ga and Na system to enhance patient access to treatments for rare and intractable conditions, while rapidly designating essential medicines to establish a stable domestic supply chain.

Once an item is designated as an orphan drug, it becomes eligible for expedited review during the marketing authorization process. It receives various commercial and regulatory incentives, including a specified period of post-marketing exclusivity. Furthermore, the submission requirements for regulatory approval are streamlined, enabling pathways such as conditional approval and waivers for local bridging data, and an expedited evaluation process is implemented through priority review. Additionally, reductions or exemptions of regulatory application fees are provided.