An Italian pharmaceutical group’s decision to deregister its wholly owned Chinese affiliate has abruptly removed an oral therapy for Cushing’s syndrome from the mainland market, underscoring systemic weaknesses in China’s rare‑disease drug ecosystem. Recordati’s Chinese arm, RuiKangDi (锐康迪), has formally filed to cancel its registration, a move that will end local supply of several orphan medicines including the sole oral treatment for Cushing’s available in China, osilodrostat (奥唑司他).
Osilodrostat was approved in China in September 2024 and launched commercially in April 2025, yet its on‑market life has lasted less than a year. The medicine had been used earlier under pilot programmes in Boao since 2021, but limited commercial uptake and the high cost of patient support and market development appear to have contributed to Recordati’s decision to withdraw.
For patients with Cushing’s syndrome, the withdrawal is more than an inconvenience: clinicians and patient groups describe osilodrostat as a “life‑saving” oral option for managing hypercortisolism. One patient who told media they had taken the drug for four years said their out‑of‑pocket spending exceeded RMB 1 million (roughly US$135,000), a striking illustration of both the clinical dependence some patients have on orphan medicines and the financial burdens they bear.
Pricing and reimbursement dynamics help explain why sales were so thin. The drug sold in China at about RMB 8,000 per box (roughly US$1,100) and industry estimates put annual treatment costs at around RMB 200,000 (about US$27,000). Osilodrostat passed an initial screening for potential inclusion on China’s national reimbursement list in August 2025, with experts anticipating that coverage could slash patient co‑payments to below RMB 2,000 per month; ultimately, however, it and two other Recordati products failed to be listed.
The commercial picture for rare‑disease medicines in China is stark: high development costs, tiny patient populations and fragile multi‑tier payment systems translate into weak revenues even for clinically important drugs. Analysts put the number of patients who actually bought osilodrostat through domestic pharmacies at only a few dozen, and small specialist firms that focus on orphan drugs report steep burdens from market education, patient support programmes and limited cash flow.
Recordati is not the only company to pull back. In 2024 BioMarin withdrew its enzyme replacement therapy for mucopolysaccharidosis IVA from China, leaving patients with no licensed alternative, and supply disruptions have afflicted other orphan drugs — including a widely used treatment for tyrosinaemia type I that fell into shortage last year despite being on the reimbursement list and off‑patent.
The immediate consequence of these commercial dynamics is human: patients face the risk of interrupted therapy and the loss of scarce treatment options. The deeper policy question is how China — and other markets with evolving insurance schemes — will create incentives for sustained supply, whether through reimbursement reform, guaranteed procurement, local manufacturing incentives or other mechanisms that reduce commercial risk for suppliers while protecting public health.
If China wishes to make rare‑disease care reliable, it must tackle both payment and supply. Stronger multi‑payer arrangements, more predictable pricing and procurement signals to manufacturers, and measures to encourage domestic production of orphan drugs would lower the chance that individual corporate decisions translate into national shortages. Absent such steps, the market will continue to deliver spotty access to important but unprofitable medicines, with patients bearing the human and financial costs.