EMA Recommends Conditional Approval for First Tissue-Agnostic Cancer Treatment

Regulatory NewsRegulatory News | 26 July 2019 |  By 

The European Medicines Agency’s (EMA) human medicines committee (CHMP) on Friday recommended the conditional approval for the first medicine to treat tumors with a specific mutation, regardless of where in the body the tumor originated.  

Bayer’s Vitrakvi (larotrectinib) is as a treatment of adult and pediatric patients with solid tumors that display a Neurotrophic Tyrosine Receptor Kinase (NTRK) gene fusion, according to EMA. Treatment with Vitrakvi is recommended for patients whose disease has spread or cannot be surgically removed, and who have no other satisfactory treatment options. 

Vitrakvi’s safety and efficacy were evaluated in three single-arm trials with a total of 102 adults and children with different cancers. The conditional approval means EMA is accepting less complete data than normally expected because it believes the benefit of the medicine’s immediate availability to patients outweighs the risks of not having all the data available. 

“The share of patients who responded to treatment with Vitrakvi was 67%,” EMA said. “Of those, the response lasted six months or longer in 88% and 12 months or longer in 75%. Tumour responses were seen both in rare tumour types such as infantile fibrosarcoma and salivary gland tumours, as well as in common diseases such as lung and colon cancer.”

Bayer also noted that the incidence of TRK fusion cancer is rare overall and affects fewer than “a few thousand patients across Europe annually.”

The US Food and Drug Administration (FDA) similarly offered Vitrakvi accelerated approval in December 2018. The treatment has also received approvals in Brazil and Canada. 

In addition, FDA offered another tissue-agnostic accelerated approval for Merck’s Keytruda (pembrolizumab) in May 2017.

A month after that initial approval, a CDER official co-authored an article for ASCO on tissue-agnostic drug development, concluding: “Ultimately, the goal of drug development is to bring effective drugs that benefit patients as quickly as possible. Investigating the effects of a drug agnostic of tumor type may be one pathway for drug development; however, every drug presents unique circumstances in regard to the population of patients who might benefit from it. Furthermore, development agnostic of tumor type could actually slow drug development if there are differential effects across tumor types by diverting resources from enrolling patients in a predominant population or in the tumor type most likely to respond.”



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Tags: Bayer, EMA, FDA

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