Nicola Ambler Jan 31
EMA Hooks Bluebird and Kite with Enticing Regulatory Pathways

EMA Hooks Bluebird and Kite with Enticing Regulatory Pathways

KITE PHARMA’S RECENT ANNOUNCEMENT OF A MARKETING AUTHORISATION APPLICATION TO THE EMA FOR THEIR LEAD PRODUCT, AXICABTAGENE CILOLEUCEL, HAS RE-IGNITED EUROPE AS A CRUCIAL STRATEGIC TOOL FOR MARKET ACCESS

The EMA has recently granted access to its Priority Medicines (PRIME) regulatory initiative, which seems to be the driving force behind Kite’s application submission. The purpose of the PRIME initiative is to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based on enhanced interaction and early dialogue with developers of promising medicines, to optimise development plans and speed up evaluations so these medicines can reach patients earlier.

All of this allows the EMA to offer early and proactive support during the generation of robust data and enable accelerated assessment of applications. The end goal, as always, is to help patients to benefit as early as possible from therapies that may significantly improve their quality of life.

This is the first application for a CAR-T therapy that has been submitted to the EMA…but will it be the first to be approved?

Are Kite pharma winning the European CAR-T race?

Will it be the first ATMP blockbuster product in Europe? Possibly. The results from Kite’s ZUMA-1 trial are extremely promising with 82% of patients achieving a response rate after a single infusion of the drug and a further 44% of patients in an ongoing response after a median follow-up of 8.7 months.

 

Less than a week later, Bluebird Bio announce they're planning to bring their gene therapies to market in Europe before the U.S., as a result of the EMA’s more flexible and favourable regulatory pathways, specifically, the adaptive pathways process. Adaptive pathways are described by the European Medicines Agency as, “a scientific concept for medicine development and data generation which allows for early and progressive patient access to a medicine.”

 

Bluebird's head of Europe, Andrew Obenshain, told the Daily Telegraph that the company is already in negotiations with the EMA and the U.K.'s Medicine and Healthcare products Regulatory Agency (MHRA) on possible regulatory filings.

 

New regulatory frameworks and increased openness from agencies demonstrate just how progressive the EU regulatory environment is in terms of granting market authorization for advanced therapies. What’s more, Kite Pharma’s submission is an indication of how rapidly this market is maturing and hurtling towards commercialisation. We spoke to Kite’s Adrian Bot at the Phacilitate Leaders’ Forum back in 2016 about their barriers to commercialisation and what they were doing to overcome these. Here’s an excerpt from the interview with Adrian: