A new investigation reveals US regulators had concerns about Ocrevus effectiveness in women with primary progressive multiple sclerosis, yet approved the treatment for both sexes.
A major medical investigation has questioned whether one of the world’s most expensive multiple sclerosis treatments may be doing more harm than good for women patients, despite generating billions in sales for pharmaceutical giant Roche.
The BMJ probe uncovered internal US Food and Drug Administration documents showing regulators had serious reservations about Ocrevus (ocrelizumab) when approving it for primary progressive MS in 2017. According to the investigation, FDA reviewers noted “near zero efficacy” in some patients and flagged data integrity issues at clinical trial sites.
The Approval Concerns
Documents show the FDA identified multiple red flags during the approval process. The drug showed no benefit in preventing disability progression among women in the key ORATORIO trial, which formed the basis for regulatory approval. Yet Ocrevus was approved for both men and women without seeking input from an external advisory committee.
Manufacturing quality problems also emerged during the review process. But perhaps most concerning was an unusual imbalance in breast cancer cases among trial participants, prompting ongoing safety monitoring that won’t conclude until late 2030.
The Financial Stakes
Ocrevus costs around $80,000 annually in the US – roughly £60,000 at current exchange rates. The drug has become Roche’s top-selling treatment, generating billions in global revenue since its approval.
Primary progressive MS affects about 15% of the estimated 130,000 people living with multiple sclerosis in the UK. Unlike relapsing forms of the condition, PPMS involves steadily worsening symptoms without periods of recovery.
A petition now before the FDA seeks revocation of Ocrevus approval for PPMS, arguing safety risks outweigh benefits chiefly for women. The petition alleges the approval was flawed and potentially harmful.
The Industry Response
Roche maintains that long-term data spanning up to 10 years demonstrates sustained effectiveness in reducing disability progression. The company points to safety analyses showing no excess breast cancer risk compared to the general MS population.
Dr Sarah Matthews, a consultant neurologist, said: “These findings highlight the importance of individualised treatment decisions based on careful risk-benefit analysis for each patient.”
Source: @bmj_latest
Key Takeaways
- BMJ investigation reveals FDA concerns about Ocrevus effectiveness in women with primary progressive MS
- Drug costs around £60,000 annually but showed no disability progression benefit in women during trials
- Petition seeks FDA approval revocation citing safety risks outweighing benefits
What This Means for Kent Residents
Kent residents with primary progressive MS can access Ocrevus through NHS Kent and Medway Integrated Care Board, but only with evidence of active inflammatory disease. Local neurologists at East Kent Hospitals University NHS Foundation Trust and Maidstone and Tunbridge Wells NHS Trust assess patient eligibility based on strict NICE criteria. Patients currently receiving Ocrevus or considering treatment should discuss these findings with their GP or MS specialist nurse to ensure their individual risk-benefit profile remains appropriate, especially regarding breast cancer monitoring for women.
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