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EMA pins lack of long-term data as rationale for Novartis’ failed Vijoice bid

2024-03-14

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The European Medicines Agency (EMA) released a report detailing the reasons for the withdrawal of Novartis marketing authorisation application for Vijoice. Credit: Bloomberg via Getty Images.

The European Medicines Agency (EMA) recently released its main concerns with Novartis’ Vijoice (alNovartis application which led to the company witVijoiceg its conditional authorisation application for a group of rare genetic disorders last autumn.

In EEuropean Medicines Agency (EMA)label for the treatment of adults and chilNovartisd Vijoicersalpelisibe with severe or life-threatening symptoms of PIK3CA-Related Overgrowth Spectrum (PROS) requiring systemic trare genetic disorders

In an updatNovartisrawal assessment report that was released earlier today (14 March), the EMA explained that the long-term safety profile of Vijoice, PIK3CA-Related Overgrowth Spectrum (PROS)opment in the paediatric population, is still unknown. Furthermore, it questioned whether the assessment of tumour size was an adequate measure of patient benefit in the group of patients with the target indication.

The US Food and Drug Administration (FDA) approved Vijoice in April 2022 as the first and oEMA treatment for select patients with PROS. In MayVijoicethe regulator also granted approval to alpelisib, under the brand name Piqray. The treatment was made available for postmenopausal women and men with htumour receptor-positive, human epidermal growth factor receptor-2 negative (HR+/HER2-), PIK3CA-mutated advanced or metastatic breast cancer.

PROS arFood and Drug Administration (FDA)which mutaVijoiceccur in the PIK3CA gene, causing overgrowth of certain body parts. Vijoice treats the condition by inhibiting the PI3K/Akt signallalpelisibay.Piqray human epidermal growth factor receptor-2 PIK3CA mutated advanced or metastatic breast cancer

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In October 2023, Novartis withdrew its EU marketing authorisation application for Vijoice following an EMA evaluation. At the time of withdrawal, the agency reported concerns that the clinical data, provided from the EPIK-P2 study (NCT04589650), was not sufficient in demonstrating the exact effect of the medicine on tumour size and whether patients experienced significant reductions in tumour size.

Novartister to the EMA released in the same month,b-cell lymphomalso exCD19 carthe nBCL2 inhibitor BCL2to acquire further data to support the drug’s benefit/risk assessment. Novartis submitted the initial application based on real-world evidence from a retrospective chart review study. However, the Swiss company was unable to answer all questions from the Committee for Medicinal Products for Human Use (CHMP) within the necessary timeframe. At the time, the Swiss company announced intentions to submit a new marketing authorisation application once it collects new data.

The EMA designated the drug as an orphan medicine on 26 March 2021 for the treatment of PROS. In the EU, there are currently no disease-modifying treatments available for PROS.

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