Novartis' data controversy
The FDA has suggested that some manipulated data was used as part of Novartis' regulatory filing for Zolgensma. The spinal muscular atrophy (SMA) gene therapy was approved by the FDA in May.
The integrity of Zolgensma's clinical profile does not appear to be in doubt, with the FDA noting that "the totality of the evidence demonstrating the product's effectiveness and its safety profile continues to provide compelling evidence supporting an overall favourable benefit-risk profile." There is no suggestion, therefore, that approval will be revoked.
For Novartis, however, the optics are less favourable; particularly as the Swiss company has conceded that it was aware of the data manipulation issues before Zolgensma was approved. CEO Vas Narasimhan's earlier pledge to rebuild trust between the pharma industry and society provides an awkward backdrop to proceedings.
Dupixent gains more label momentum in younger patients
Sanofi and Regeneron Pharmaceuticals continue to build out the commercial and clinical profile of their blockbuster atopic dermatitis treatment Dupixent. In Europe, it has now been approved for use in adolescent patients (aged 12 to 17), mirroring an indication cleared by the FDA in March.
Sanofi and Regeneron also confirmed positive Phase III data for Dupixent in moderate-to-severe patients aged 6 to 11.
In response, we have snap-polled 50 US-based dermatologists to get a better understanding of how they may use Dupixent in this age group. Results will be published on Friday (9 August).
Neurologists weigh in on Biogen's Vumerity data
Biogen is hoping to grow its multiple sclerosis franchise by launching Vumerity, a follow-up to its hugely successful oral treatment Tecfidera; which also faces the prospect of losing patent exclusivity in the next few years.
Biogen and its development partner Alkermes last week announced positive head-to-head gastrointestinal tolerability data for Vumerity versus Tecfidera, showing the former to be a potentially more patient-friendly treatment option.
We ran the data past 85 neurologists based in the US and EU3 (France, Germany and the UK) who suggested that on average they would expect to switch nearly four in 10 of their existing Tecfidera patients to Vumerity.
KOL impressed with Keytruda in TNBC
Merck & Co. recently announced exciting top-line data for its PD-1 inhibitor Keytruda in combination with chemotherapy in triple-negative breast cancer (TNBC); notable not only as this is a relatively new indication for immunotherapy, but also as Merck evaluated Keytruda as a neoadjuvant therapy in patients regardless of PD-L1 status and used pathological complete response (pCR) rate as the study's primary endpoint.
We spoke to a leading breast cancer key opinion leader (KOL) this week who told FirstWord that he is hoping to see a 15% improvement in pCR for Keytruda plus chemotherapy versus chemotherapy alone when detailed results from the Keynote-522 study are presented later this year. This pCR benefit is highly likely to translate into prolonged disease-free survival (DFS), he adds. More here
KOL suggests way forward for Entresto in recently missed indication
One expert told FirstWord this week that Novartis may be advised to run a number of smaller studies for Entresto in patient subgroups shown to have a more active response to the therapy. Novartis recently confirmed that its Phase III PARAGON-HF study - evaluating Entresto as a treatment for heart failure with preserved ejection fraction (HFpEF) - narrowly missed its primary endpoint. Approval in this indication would effectively double the number of patients eligible for treatment with Entresto, which generated global sales of $778 million in the first half of 2019.
Novartis plans to share data from PARAGON-HF with regulators, though the KOL we spoke to suggested it very unlikely that Entresto will be approved for HFpEF based on these study results alone. More here.
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