Brain cancer vaccine wins first UK 'innovative medicine' status...

A potential new cancer treatment has become the first therapeutic to be awarded the new ‘Promising Innovative Medicine’ (PIM) designation, which could lead to fast-track access to patients.

The Early Access to Medicines Scheme (EAMS) is a new scheme launched by the UK Medicines and Healthcare Products Regulatory Agency (MHRA) in April this year. It is intended to allow patients with ‘life threatening or seriously debilitating’ conditions access to potentially useful new medicines ahead of formal approval for their use, which can take several years. The scheme is intended to benefit both seriously ill patients who lack other viable treatment options, and companies developing new medicines who could find the designation helpful in attracting investment.

The news does not mean that patients will be able to receive the therapy yet, however. PIM designation is the first of two steps in the process indicating potential eligibility for the EAMS agreed by an MHRA scientific meeting and based on preliminary clinical trial data. The MHRA assesses whether or not the product:

• is for a serious condition with high unmet clinical need
• is likely to offer a major advantage over current treatments
• has potential benefits that outweigh the potential harms

The second stage is an Easy Access to Medicines Scientific Opinion from the MHRA, which describes the available data, the risks and benefits of the new medicine and allows a decision to be made on whether it is eligible for early access by selected patients ahead of licensing.

The new medicine, DCVax-L, has been developed by Northwest Biotherapeutics Inc. who will continue with clinical development and hope to accrue appropriate data to allow progression to an MHRA Scientific Opinion on eligibility for the EAMS. DCVax-L is for the treatment of new and recurrent malignant gliomas and is not a classical drug but a cellular or biological therapy – in this instance what is termed a cancer vaccineintended to prime the body’s own immune defences to target and destroy tumour cells.

NorthWest’s Chief Executive Linda Powers said that the scheme offered“a very practical balance between clinical benefits and risks, through careful scientific evaluations”.

Life Sciences Minister George Freeman commented: “We want to make Britain the best place in the world to design and deliver 21st Century healthcare technologies which is central to our life science strategy. This Promising Innovative Medicine designation is the first crucial step in accelerating access to new medicines, giving real hope to patients and their families”. He also said he hoped to see more companies of all sizes and medical charities nominating products for the EAMS.

US FDA grants fast track status to Pfizer’s clostridium difficile vaccine candidate

The US Food and Drug Administration (FDA) has granted Fast Track designation to the Pfizer’s investigational Clostridium difficile (C. difficile) vaccine candidate (PF-06425090). Currently in phase 2 clinical development, the vaccine candidate is designed to prevent C. difficile-associated disease, which can include life-threatening diarrhoea and pseudomembranous colitis.
 
“C. difficile is a growing, difficult-to-treat healthcare-associated infection,” said Dr. Emilio Emini, senior vice president of vaccine research and development for Pfizer. “No vaccine is currently available to prevent the infection-associated disease. In the United States alone, there are approximately 250,000 cases of C. difficile-associated disease, resulting in approximately 14,000 deaths each year.”
 
The FDA’s Fast Track approach is a process designed to facilitate the development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address an unmet medical need.
 
Clostridium difficile (klos-TRID-e-um dif-uh-SEEL), often called C. difficile, is the most frequent cause of healthcare-associated infections. C. difficile is a spore-forming, Gram-positive anaerobic bacillus that produces two exotoxins: toxin A and toxin B. It is a common cause of antibiotic-associated diarrhoea (AAD) and accounts for 15-25 per cent of all episodes of AAD.
 
Illness from C. difficile most commonly affects older adults in hospitals or in long-term care facilities and typically occurs after use of antibiotic medications. However, studies show increasing rates of C. difficile infection among people traditionally not considered high risk, such as younger and healthy individuals without a history of antibiotic use or exposure to healthcare facilities.
 
Infection with C. difficile places a significant burden on healthcare facilities and has been shown to substantially increase hospital costs, hospital length of stay, and contribute to mortality.