NETHERLANDS —The European Medicines Agency’s committee for medicinal products for human use (CHMP) has put forth a recommendation to revoke the marketing authorization for Novartis’ treatment for a painful complication associated with sickle cell disease.
This decision comes after a recent study failed to confirm the clinical benefit of the drug.
The review conducted by CHMP focused on the outcomes of the STAND study, which revealed that Adakveo (crizanlizumab) did not decrease the number of painful crises leading to healthcare visits.
Surprisingly, patients treated with Adakveo experienced slightly more painful crises on average, resulting in subsequent healthcare visits compared to those who received a placebo.
This review aligns with the findings from a data release by Novartis in January, which also indicated no statistically significant difference between the drug and a placebo in terms of the annualized rates of vaso-occlusive crises that necessitate a healthcare visit within the first year of treatment.
Regarding safety, the STAND study did not raise new concerns but did demonstrate a higher incidence of severe and serious treatment-related side effects with Adakveo compared to the placebo.
Consequently, CHMP concluded that the potential benefits of the drug do not outweigh the associated risks.
As a result, the agency has advised healthcare providers not to initiate new treatments with Adakveo.
Novartis responded to the recommendation by acknowledging the failure of the STAND study to fulfill the specific obligations of the drug’s conditional marketing authorization.
However, the company expressed disappointment with the recommendation to revoke the authorization in the European Union.
The future course of action by the US Food and Drug Administration (FDA), which approved Adakveo in 2019, remains undisclosed.
In its annual report for 2022, Novartis disclosed that Adakveo generated US$200 million in sales and had reached over 11,800 patients with vaso-occlusive crises caused by sickle cell disease worldwide.
Additional CHMP decisions
In addition to the revocation of Adakveo’s authorization, CHMP made other notable recommendations.
Bristol Myers Squibb’s cancer drug Opdivo (nivolumab) received a positive opinion for label expansion to include neoadjuvant treatment of non-small cell lung cancer (NSCLC).
The recommendation was based on the positive outcomes of the CheckMate-816 trial, which demonstrated a significant improvement in event-free survival and pathologic complete response when Opdivo was administered in combination with chemotherapy prior to surgery.
Furthermore, CHMP recommended the approval of Marinus Pharmaceuticals’ Ztalmy (ganaxolone) for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2 to 17 years.
If granted authorization by the European Commission, Ztalmy will become the first treatment in the EU indicated for the adjunctive treatment of seizures associated with CDD.
Orion Corporation will be responsible for its commercialization. Ztalmy received approval in the United States in March 2022.
However, Ipsen Pharma’s Sohonos (palovarotene), intended for the treatment of the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), faced a setback as CHMP rejected its application for the second time.
Ipsen expressed disappointment but reiterated its commitment to finding a new treatment option for the FOP community.
CHMP also noted two withdrawals of applications: Otsuka withdrew its application for marketing authorization of Asimtufii for the maintenance treatment of schizophrenia, citing a change in the company’s strategy.
Similarly, Roche withdrew its application for Susvimo, a treatment for wet age-related macular degeneration, due to device-related complications.
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