Bluebird's EU exit over a German pricing row left CALD children without Skysona

Darius, a Spanish teenager, was among the first children in the world cured of early cerebral adrenoleukodystrophy (CALD) — a rare, fatal hereditary disease that destroys the brain's myelin in previously healthy children aged five to eight — thanks to Skysona, a gene therapy he received in the research phase at age five. His elder brother Danuk had died of the same illness at ten months. Since 2021, no child in Europe has been able to get Skysona commercially.

The reason is commercial, not medical. Skysona was approved by the EMA in July 2021, but its maker, US biotech Bluebird Bio, had spent two years fighting European reimbursement authorities over Zynteglo, another gene therapy. After German hospitals did not reproduce the trial results, the government sought to cut Zynteglo's 1.6-million-euro price to 600,000-700,000. In August 2021 Bluebird announced it was shutting its European operations, and Skysona's pricing talks — which had not even started — never began.

On November 18, 2021, the EMA formalised Bluebird's 'decision to permanently discontinue the marketing of the product for commercial reasons'. 'It would cost Bluebird more to deliver these therapies than what European authorities were willing to pay,' a spokesperson said. Bluebird pivoted to the US, where both therapies were approved in summer 2022 — $2.8 million for Zynteglo, $3 million for Skysona. 'Now all we can tell the families is to raise $3 million and try to go to the United States,' said geneticist Aurora Pujol.

Dozens of European children with early cerebral adrenoleukodystrophy lost access to the region's only approved treatment for the fatal disease.

Germany is the launch market most EU countries reference, so a renegotiated low price would have undercut the therapy's economics across Europe.

Ultra-rare-disease therapies sell in tiny volumes, making EU market access hard to justify at prices authorities would accept.

Skysona was collateral: the exit decision was driven by the Zynteglo pricing fight, but both therapies left the EU together.

European governments had never begun negotiating Skysona financing, so no country had leverage to keep it on the market.

Walking away from one country's pricing regime can close a whole region: in ultra-rare diseases, a commercial dispute strands exactly the patients the drug was built for.

As of the November 2023 report, dozens of European children with CALD had missed the treatment window since the 2021 withdrawal; the therapy's own research roots were European, begun at INSERM's public hospitals and helped by Spain's ELA patient association, which sent three children to the first 2006 Paris trial. The association called the situation 'incomprehensible and unbearable': 'We are back to how we were 20 years ago, watching children die.'