- A key committee of the European Medicines Agency last week recommended approval for two orphan drugs from Biogen and BioMarin Pharmaceutical which target rare neurodegenerative diseases in children.
- Biogen's drug Spinraza (nusinersen), already approved in the U.S., treats spinal muscular atrophy, while BioMarin's medicine Brineura (cerliponase alfa) is designed to slow or halt progression of a form of Batten disease known as CLN2.
- The EMA's endorsement sends the companies' applications for approval of the drugs to the European Commission, which is expected to grant a final approval within the next few months. Approval was also recommended for nine other medicines, including three biosimilars.
Brineura will likely be BioMarin's sixth approved drug, provided the EC follows the EMA's decision and grants approval later this quarter.
In the U.S., the Food and Drug Administration is currently reviewing Brineura and a decision is expected Thursday.
Securing approval is important for BioMarin, which last year shuttered its development program for drugs targeting Duchenne muscular dystrophy. The decision came after discussions with the EMA indicated to BioMarin its lead drug Kyndrisa (drisapersen) would likely be rejected. Europe had been BioMarin's last hope for the closely watched drug following an FDA denial in January 2016.
Brineura would be the first drug from the company approved since that costly setback. BioMarin's application was supported by evidence from an open-label trial that tested the drug in 24 children plus additional data from an extension of that study. Since CLN2 is an ultra rare disease, the EMA approved the drug under exceptional circumstances and will require BioMarin to further test Brineura's safety and efficacy.
For Biogen, the EMA recommendation for approval of Spinraza sets up a broader market to build on the drug's launch in the U.S. If the EC agrees to license the drug for marketing, Biogen will then have to negotiate pricing and reimbursement with each member state government.
Spinraza's high price tag — $750,000 for the first year of treatment, and $375,000 annually thereafter — has slowed uptake of the drug despite being the first treatment approved for SMA. Anthem and Humana, two major U.S. insurers, have placed limits on coverage for the drug. While Europe's insurance market is markedly different from the U.S., Biogen will still likely face challenges in any future price negotiations with E.U. member states.
On the other end of the drug development spectrum from novel rare disease drugs, the EMA also endorsed three biosimilar drugs: two versions of Roche's Rituxan (rituximab) from Novartis' Sandoz unit and a biosimilar of Amgen's Enbrel (etanercept), also from Sandoz.
Novartis has made a major commitment to biosimilar development through Sandoz, and expects to launch three other biosimilars of oncology and immunology biologic drugs by 2020. Novartis has already secured two approvals for copies of Enbrel and Neupogen (filgrastim), another drug made by Amgen, in the U.S.
Elsewhere, the EMA recommended approval of Sanofi and Regeneron's arthritis hopeful Kevzara (sarilumab). The drug is also under review in the U.S.