The primary treatment for the rare bleeding disorder hereditary factor X deficiency has now been initiated across the UK.

The UK is the first country in Europe where Coagadex will be made available, via a Commercial Medicines Unit tender, which starts on July 1st.

Bio Products Laboratory’s Coagadex was accepted in Europe in March for the treatment and prophylaxis of bleeding as well as for perioperative management in patients with this state.

According to Dr Steve Austin, director of St George’s University Hospitals NHS Foundation Trust, London “The prior focus has been on treating Factor X deficiency with blood infusions of plasma. For the first time, patients with this exceptional bleeding disorder can receive a definite factor X replacement that has been proven useful and successful in clinical studies.”

Hereditary factor X deficiency has an impact around 700 patients in Europe, and is described by a lack of factor X protein in the blood, which plays a critical role in coagulation.

People with this insufficiency are at bigger risk of bleeding, including inside the brain, lungs or gastrointestinal tract in the most severe cases, which can be life-threatening.

The CMU works on behalf of the Department of Health and the NHS and in corporation with the people who buy pharmaceuticals for hospitals across the NHS in the UK.

Date:  7th June 2016

http://www.pharmatimes.com/news/uk_launch_for_first_hereditary_factor_x_deficiency_treatment_1035301

The European Medicines Agency (EMA) today published a report from a multi-stakeholder expert meeting held on 27 May 2016 to look at feasible ways to promote the advancement of ATMPs in Europe and increase patients’ access to these new treatments.

ATMPs include:

• Gene therapies
• Tissue engineered products
• Somatic cell therapies

 These medicines have potential to reshape the treatment of a wide range of conditions, mainly in disease areas where predictable approaches are insufficient. However, eight years since EU legislation on ATMPs entered into force in 2008, only five ATMPs are currently sanctioned.

According to EMA’s Executive Director Guido Rasi, ‘We have organized this meeting with all relevant stakeholders to discuss concrete proposals on how we can nurture a regulatory environment that encourages development of ATMPs, safeguards public health and, ultimately, facilitates timely access for patients to much needed treatments.’

The meeting brought together leading academics and researchers, representatives from patients’ and healthcare professionals’ organizations, small and large pharmaceutical companies, the investment community, incubators and consortium organizations, health technology assessment (HTA) bodies, national competent authorities and the European Commission. In their discussions they focused on four key areas:

• Assisting research and development areas.
• Optimizing regulatory processes for ATMPs.
• Moving from hospital release to marketing approval.
• Improving funding, investment and patient access.

The main ideas and solutions projected by the different stakeholders are sum up in the meeting report. EMA and its scientific committees, together with the European Commission and the national knowledgeable authorities, have started conversing the proposals made during the meeting. Tangible actions will be determined over the next few months and shared with stakeholders.

Date: 03/06/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/06/news_detail_002543.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has started a appraisal of the plan that describe first-in-human clinical trials and the data needed to allow their suitable design and allow instigation. This is being done in collaboration with the European Commission and the Member States of the European Union (EU).

The evaluation will recognize which areas may need to be revised in the light of the tragic incident which took place during a Phase I first-in-human clinical trial in Rennes, France, in January 2016. The trial led to the death of one participant and hospitalization of five others.

EMA’s assessment will take into relation the findings from two in detail investigations into what went wrong during this check, one carried out by the provisional Specialist Scientific Committee (TSSC) set up by the French medicines agency ANSM and the other by the assessment générale des affaires sociales (IGAS), the inspectorate for social affairs in France.

Both reports comprise a sequence of recommendations concerning the requirements for sanction and ways of first-in-human clinical trials for further examination by the international regulatory and public health community.

EMA’s work will be the hub on best practices and direction. The objective is to agree a concept paper by July identifying areas for change and proposals to further reduce the risk of similar accidents. The concept paper will form the basis for an EU-wide review of the guidelines. This process will comprise of targeted discussions with stakeholders and a public discussion on anticipated changes shortly in 2016.

The EMA appraisal has started with two groups of experts who are carrying out preliminary work. One group is looking at pre-clinical aspects and the data needed from laboratory tests or animal studies to safely start first tests in humans. The other group is looking at clinical aspects of the design of first-in-human trials and how these could be improved to better ensure the safety of human volunteers taking part in these trials. This will lead into one EU-wide expert group discussion on review of guiding principle.

Clinical trials are vital for the improvement of medicines and without them patients cannot expand contact to new potentially life-saving medicines. In the EU, the approval and conduct of clinical trials is within the remit of the relevant authorities of the European Member States.

EU guidelines are in place to ensure that these clinical trials are conducted as safely as possible.

Severe adverse reactions in healthy volunteers such as those experiential in the trial in Rennes are extremely rare during clinical trials. Since 2005, approximately 14,700 phase I clinical trials (with participation of 305,000 subjects) have been conducted in the EU, including 3,100 first-in-human studies. Only one other rigorous incident has been previously reported in that time in the EU

Date: 27/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002538.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has suggested the conceding of marketing authorizations in the European Union (EU) for two new permutation therapies against chronic hepatitis C virus (HCV) infection, Epclusa and Zepatier.

HCV infection is a foremost European public health confront. It affects between 0.4% and 3.5% of the population.

Epclusa and Zepatier are from new generation of medicines for chronic HCV disease, direct-acting antivirals, that give high rates of cure of HCV infection and that have, in the past few years, reform the way this disease is treated. These medicines obstruct the action of proteins which are necessary for viral imitation. Epclusa targets the proteins NS5B and NS5A, while Zepatier targets the proteins NS3/4A and NS5A.

These new treatment allow cure of patients with chronic HCV infection without the need for interferon medicines which are linked with poor acceptability and severe side effects. In spite of the rapid development of new therapies, including interferon-free regimens, doctors and patients can still gain advantage from substitute treatment options.

Epclusa contains sofosbuvir, already approved under the name Sovaldi and as a combination therapy with ledipasvir under the name Harvoni, and velpatasvir which is a novel HCV protein inhibitor. The safety and effectiveness of Epclusa was tested in clinical trials involving over 2,000 patients, to review that the HCV was no longer detected in the blood 12 weeks after the end of treatment with or without ribavirin. Majority of the patients across all genotypes had no evident virus in their blood 12 weeks after the end of the treatment and could therefore be considered to be cured of their HCV infection. The most common side effects reported in clinical trials were headache, fatigue and nausea.

Zepatier contains two novel HCV protein inhibitors, grazoprevir and elbasvir. This fixed-dose combination of direct-acting antivirals targets genotypes 1 and 4 of the disease.

The effectiveness and protection of Zepatier was tested in clinical trials involving approximately 2,000 patients. The medicine also showed a persistent virologic response. The safety of the medicine was also measured approving and the most common side effects reported in clinical trials were fatigue, headache and nausea.

Date: 27/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002537.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency’s (EMA) 2015 annual report published emphases on the Agency’s central chores which are basically:

• Assessment of medicines
• Promoting research
• Development of new and advanced treatments
• Checking of the benefits and risks of medicines in real life

In 2015, the Agency suggested marketing endorsement for 93 medicines for human use, which comprise 39 new active ingredients, and 14 medicines for veterinary use, containing seven new active elements.

About one in two applicants who received a positive view for their medicine had received scientific guidance from EMA during the progress phase of their product; this figure rises to 85% for medicines containing a new active ingredient. Scientific guidance is EMA’s key tool to stimulate the collection of high quality data on the advantages and risks of medicines.

This is a positive trend showing that patients increasingly make use of the opportunity to directly report on side effects they experience. For human medicines, more than 1.2 million reports on suspected side effects were recorded. Over 48,000 reports instigated from patients in the European Economic Area, a 30% rise compared to 2014.

In 2015, the product information on many medicines was modernized as a new safety information had become available.

EMA’s annual report also shows some of the main projects, initiatives and achievements in 2015 that have had an important influence on the Agency and the way it functions. These are:

The preparation for the launch of Priority medicines to the maintenance and the advancement of medicines that address unmet medical needs

Initiatives to help fight antimicrobial resistance and other public health threats such as Ebola as well as the execution of various new pieces of legislation including a range of new activities to further support the safety checking of medicines.

The report also covers three interviews with stakeholders and EMA representatives on topics of major interest in the area of medicines and health in 2015, comprising:

• Best way of alignment for data requirements from regulators and health-technology-assessment (HTA) bodies.
• The way immunotherapies bring new hope to cancer patients.
• Use of big data in healthcare is challenging or an opportunity.

Date: 17/05/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/05/news_detail_002531.jsp&mid=WC0b01ac058004d5c1

The rising and emergent issue of high medicine prices and its effect on the sustainability of health care systems is getting more and more consideration in many countries around the world. Regulators are ready to play their part in solving the crisis and in facilitating sustained access of patients to safe and useful medicines.

In the article published in the New England Journal of Medicine (NEJM) two representatives of the European Medicines Agency (EMA), i.e. its Executive Director and Senior Medical Officer, as well as Heads of two national agencies discuss possible regulatory interventions.

The pricing of medicines is clearly out of their dispatch, medicine regulators cannot overlook the recent debate on the cost of medicines and can make a role to reasonably priced care, give details the authors in their article entitled ‘Drug regulation and pricing – can regulators influence affordability?

The article, co-authored by EMA’s Executive Director Guido Rasi, its Senior Medical Officer Hans-Georg Eichler, the Executive Director of the Dutch Medicines Evaluation Board Hugo Hurts and the president of the German Federal Institute for Drugs and Medical Devices Karl Broich, is available in open access in the NEJM.

According to the authors of the article, there are five main ways European regulators can help:

• Facilitate the quick sanction of generics and biosimilars, as this facilitate competition and drives down prices;
• Works to guarantee ‘me-too’ products (medicines comparable to already approved options) keep on coming on the market at reasonable speed, again to drive down prices through augmented competition.
• Support companies to carry out clinical trials that both assure the needs of regulators (i.e. make obvious quality, safety and effectiveness of the medicine) as well as the health-technology-assessment bodies(i.e. support the revelation of the value once the medicine is authorized, to guide payers in their reimbursement decisions).
• Assist the collection of other data that are important for payers by taking their needs into account when asking companies to conduct post-approval studies.
• Maintain higher competence of research and development in the area of medicines: by development a better model of medicines, it is anticipated that companies would potentially be able to decrease the price of their medicines. This could also mean reflecting on new approaches to medicines’ development, such as the adaptive pathways approach that is being explored by EMA.

Date: 12/05/2016

http://www.ema.europa.eu/ema/

According to a study published on May 1, 2016 in the journal Cancer Research, Hsp90 inhibitor could provide useful treatment for advanced prostate cancers that have become resistant to hormone treatment.

It will work by decreasing the production of androgen receptor modification by using latest and new method of action.

Basically prostate tumors depends on androgens to grow and extend, depriving androgen receptors of these hormones can be an efficient and successful treatment

Base on this study, researchers targeted the one of this androgen receptor alternative, AR-V7. They produced the cancer cells in the lab and injected them into mice. Then they administered a drug (onalespib) that inhibits Hsp90, a chaperone molecule involved in the transcriptional action of these receptors.

According to the study co-leader Johann de Bono, MD, PhD, MSc, Professor of Experimental Cancer Medicine at the Institute of Cancer Research (ICR), in London, United Kingdom, “We have established for the first time that Hsp90 inhibitors that can obstruct the growth of the most common abnormal androgen receptors that cause many prostate cancers to stop responding to existing treatments. The researchers revealed that inhibiting Hsp90 decreased the production of AR-V7, but not by disrupted its known chaperone function”.

According to the opinion of study co-leader Paul Workman, PhD, Chief Executive of the ICR, “Our study has found that Hsp90 inhibition can particularly stop resistance to hormone treatments in prostate cancer through a totally new method linking the dispensation of messenger RNA.”

Dr. Workman explained, ‘We identify Hsp90 inhibitors ‘network drugs’ because they deal with several of the signals that are hijacked in cancer all at once, across a complex network rather than just a single way,”.

Hsp90 inhibitors are already in clinical trials for numerous types of cancer.

2^nd May 2016

http://www.mdlinx.com/pharmacy/article/414

The European Medicines Agency (EMA) has recommended conceding a marketing authorization in the European Union (EU) for Zavicefta (ceftazidime/avibactam), a new treatment against multi-drug resistant bacteria.

Due to lack of availability of medicines to treat patients with infections by resistant bacteria has become a major problem in past years. It is predictable that 25,000 patients in the EU die each year from infections due to bacteria that are resistant to many medicines.

Zavicefta is a fixed combination of avibactam, a new beta-lactamase inhibitor, and ceftazidime, an antibiotic belonging to the class of third generation cephalosporins that is already accepted for use in the EU. Resistance to cephalosporins and to another class of antibiotics, carbapenems, has been increasing lately, in particular in Gram-negative bacteria, and is of major concern. Beta-lactamases are enzymes implicated in bacterial resistance to these antibiotics. By restrain the action of these enzymes, avibactam restores the activity of ceftazidime against ceftazidime-resistant pathogens. This antibacterial agent also has activity against many of the carbapenem-resistant Enterobacteriaceae, where there is presently an unmet medical need as patients have very few options available due to resistance to treatment.

The medicine is to be used in adult patients with intra-abdominal infection, urinary tract infection, as well as pneumonia acquired in a hospital setting. It is also indicated for the treatment of adult patients with infections caused by certain Gram-negative bacteria, for which there are only restricted treatment options.

The effectiveness of Zavicefta against certain Gram-negative bacteria has been verified in the clinical trials that strengthen the authorization of the indications of intra-abdominal and urinary tract infections, and hospital-acquired pneumonia. The Committee for Medicinal Products for Human use (CHMP) considered that it is helpful to make Zavicefta accessible for patients with infections caused by Gram-negative bacteria, when they have few or no therapeutic options to fight the disease, and suggested to include treatment of these patients in the product information on the basis of a limited set of data.

EMA contributes to the European and global attempt to tackle antimicrobial resistance. A major area of activity is to generate an environment that encourages and facilitates the progress of new antimicrobials.

The outlook adopted by the CHMP at its April 2016 meeting is a conciliator step on Zavicefta’s path to patient access. Once a marketing authorization has been approved, decisions about price and repayment will take place at the level of each Member State, taking into account the potential role of this medicine in the circumstance of the national health system of that country.

Date: 29/04/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/04/news_detail_002523.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency (EMA) has suggested broaden the authorized use of Gazyvaro (obinutuzumab) to treat patients with follicular lymphoma. The medicine is to be used in amalgamation with bendamustine in patients who were treated before with chemotherapy.

Gazyvaro was first certified in the European Union (EU) in July 2014 for use in combination with chlorambucil in patients having chronic lymphocytic leukaemi and previously untreated.

Follicular lymphoma and chronic lymphocytic leukaemia are both rare types of cancer that affect certain white blood cells that fight infection, called B-lymphocytes. In follicular lymphoma, the body generates abnormal B cells that build up in lymph nodes.

Though useful treatments exist for follicular lymphoma, the disease often comes back and becomes ever more insistent and resistant to existing treatment options. Patients whose disease has become aggressive often die after one to two years.

The active substance in Gazyvaro is a monoclonal antibody that targets B-lymphocytes and triggers the death of cancer cells through the activation of the immune system.

The recommendation from EMA’s Committee for Medicinal Products for Human Use(CHMP) is based on the results of a phase III trial that compared the possessions of Gazyvaro given in permutation with bendamustine and followed by Gazyvaro as a preservation treatment, with the effects of bendamustine alone, in 321 patients with follicular lymphoma who did not respond to or whose disease grow with chemotherapy.

According to the study, patients treated with Gazyvaro in combination with bendamustine lived longer without their disease progressing as compare to patients treated with bendamustine alone.

The general side effects reported with the combination of Gazyvaro and bendamustine were constant with the known safety profiles of the individual medicines.

Because follicular lymphoma is rare, Gazyvaro was nominated as an orphan medicine in 2015. Orphan-designated medicines meet the criteria for ten years’ market exceptionalit.

The outlook adopted by the CHMP at its April 2016 meeting is an intermediary step on Gazyvaro’s path to patient contact. The CHMP opinion will now be sent to the European Commission for the acceptance of a decision on EU-wide marketing authorizations. Once the expansion of suggestion has been granted, a decision about price and compensation will take place at the level of each Member State considering the potential role/use of this medicine in the context of the national health system of that country.

Date: 29/04/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/04/news_detail_002522.jsp&mid=WC0b01ac058004d5c1

The European Medicines Agency’s Pharmacovigilance Risk Assessment Committee (PRAC) has adopted the final rules of procedure for public hearings to be held by the Committee. The rules of procedure depict the process and useful provisions for the groundwork, conduct and follow-up of public hearings.

For execution of these regulations, the European Medicines Agency (EMA) will arrange a domestic dry run exercise just to check the progression and trial of public hearings. The dry run is scheduled to take place at the PRAC meeting in July 2016. Public hearings could arrange as early as the fourth quarter of 2016, as soon as a significant topic is decided.

Public hearings are a new contrivance for EMA to connect European Union (EU) citizens in the regulation of medicines and to listen to their analysis, views, visions and practices. The pharmacovigilance legislation has given the PRAC the opportunity to hold public hearings as part of definite and useful reviews of medicines, mainly relevant to their therapeutic effects and available therapeutic substitutes, as well as the possibility and approval of planned and projected risk management.

According to Noël Wathion, EMA’s Deputy Executive Director, “Public hearings will improve the scientific decision-making process on the safety of medicines.”

Public hearings will be held on a case-by-case basis, where the Committee determines that assembling the outlook of the public would bring added value to its review.

Date: 15/04/2016

http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2016/04/news_detail_002512.jsp&mid=WC0b01ac058004d5c1