ORPHELIA Pharma's Kigabeq (vigabatrin) obtains PUMA for pediatric West syndrome in Europe
Approval of a pediatric-specific formulation through the PUMA system
The European Medicines Agency (EMA) has approved ORPHELIA Pharma's Kigabeq (vigabatrin) for a Pediatric Use Marketing Authorisation (PUMA), a designation for formulations specifically developed for pediatric use. Following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) on September 20, 2018, the European Commission (EC) finalized the approval in October of the same year, with the ninth amendment completed. PUMA is a European-specific authorization granted for reformulated versions of existing adult drugs for pediatric use. Kigabeq is a hybrid drug that demonstrated bioequivalence to Sabril (Lundbeck)'s 500mg granules, while introducing a differentiated soluble tablet formulation in 100mg and 500mg, which can be divided.
Indication and clinical positioning
Kigabeq has two approved indications. First, it is indicated as monotherapy for infantile spasms (West syndrome) in children aged 1 month to under 7 years. Second, it is approved as adjunctive therapy for resistant partial epilepsy that is not controlled by other antiepileptic drugs. West syndrome is a rare and severe form of epilepsy, affecting approximately 2 to 5 infants per 10,000 births, characterized by hypsarrhythmia on EEG and sudden psychomotor regression. Previously, ACTH (adrenocorticotropic hormone) injections and Sabril granules were the main treatment options. Kigabeq improves pediatric administration convenience by offering an oral soluble tablet suitable for both oral and non-oral (e.g., nasogastric) administration.
Competitive landscape and market environment
The infantile spasms treatment market is estimated to be worth approximately USD 185 to 454 million by 2026, with vigabatrin-based therapies holding a market share of about 34.4%. In the U.S. market, Lundbeck's Sabril and Mallinckrodt (now Endo)'s H.P. Acthar Gel (repository corticotropin) have formed a duopoly of FDA-approved treatments. In June 2024, Upsher-Smith received FDA approval for VIGAFYDE, the first immediately administrable oral solution of vigabatrin, intensifying formulation competition. In Europe, Kigabeq has secured a unique market position as a pediatric-specific formulation through PUMA.
Commercialization status in Europe
ORPHELIA Pharma, a subsidiary of Biocodex Group, granted Biocodex exclusive distribution rights in most EU countries in 2021. Following its initial launch in Germany, it expanded to the UK and France, with licensing agreements also signed for 14 European countries. As a non-listed company, sales data remains undisclosed, but its positioning as a formulation for rare pediatric diseases is advantageous in securing favorable insurance reimbursement and premium pricing.
The infantile spasms treatment market (estimated at USD 185 to 454 million by 2026) is dominated by vigabatrin-based therapies with a 34.4% market share. Kigabeq has established an exclusive position in Europe as a pediatric-specific formulation through the PUMA system. While Upsher-Smith's VIGAFYDE oral solution received FDA approval in 2024, expanding vigabatrin formulation competition globally, Kigabeq's PUMA-based data exclusivity and Biocodex's distribution network across 14 countries serve as significant entry barriers. Orphan pediatric formulations allow for high pricing relative to patient numbers, making them suitable for the profitability models of small specialty pharmaceutical companies. As ORPHELIA Pharma is a non-listed company, it serves as a benchmark case for orphan drug formulation redevelopment strategies under PUMA/505(b)(2). Meanwhile, the risk of visual field defects associated with all vigabatrin products, managed through REMS and risk management plans, remains a structural constraint on market expansion.
Source: EMA (ema)