NICE has requested further information on Forxiga (dapagliflozin) as a combination therapy option to treat type 2 diabetes after failing to recommend the product in draft guidance.

Bristol-Myers Squibb and AstraZeneca have been asked for further clarification and information after concerns were raised about trial data and the cost modelling of the drug.

Forxiga has a UK marketing authorisation in adults with type 2 diabetes mellitus. It’s used as monotherapy when diet and exercise alone do not provide adequate control and as an add-on combination therapy with other treatments, such as insulin and metformin.

During the appraisal, NICE’s independent Appraisal Committee questioned the evidence on the clinical effectiveness of Forxiga as an add-on to insulin and metformin.

The Committee noted that the trial data for the drug as an add-on therapy to insulin came from two placebo-controlled trials – one of which was only 12 weeks in duration. The evidence supplied for Forxiga as an add-on to metformin came from three clinical trials and a network meta-analysis.

Concerns were also raised by the Committee surrounding the comparisons made by BMS and AZ of the cost of Forxiga and with that of other anti-diabetic drug therapies and how these were initially made.

“Type 2 diabetes is a serious problem in the UK and it is important that there is a range of different treatment options available,” said Professor Carole Longson, Health Technology Evaluation Centre Director at NICE. “Unfortunately the Appraisal Committee is currently unable to recommend dapagliflozin, one of the options, for the treatment of this condition. They have requested further information from the manufacturer, which will be considered at the next Appraisal Committee meeting in April.”

Final guidance is expected in June 2013.

Biotechnology giant MedImmune has appointed Bahija Jallal, its current Executive Vice President for R&D, as its new President.

The former President of the Maryland-based company, Peter Greenleaf, will take over leadership of parent company AstraZeneca’s (AZ) Latin American business.

The transition accompanies the formal designation of MedImmune as the global biologics division of AZ, which acquired it for $15.6bn in 2007.

Bahija Jallal is a former researcher at the Max-Planck Institute of Biochemistry in Germany. While VP of Drug Assessment and Development at Chiron, she was headhunted by MedImmune in 2006, becoming its Head of Translational Science.

Commenting on that appointment, Jallal said: “I was struck by MedImmune’s history of innovation. And I looked at who they had on their board of directors and their executives. There were more women than you would see at other companies.”

The fortune paid by AZ to acquire MedImmune surprised many in the industry. The acquisition was followed by investment in its R&D capability, including the development of a new site in Cambridge, England, and the merger of the UK group with Cambridge Antibody Technology.

According to MedImmune spokesman Mike O’Brien, the biotech company’s commercial operation now formally belongs to AZ’s North American business, while its manufacturing operation is part of AZ’s global operations.

“The driver for these changes is not cost but even faster decision-making in key areas of the business and a need to reduce complexity,” he said.

Through MedImmune, AZ plans to deliver an average of one new biologic drug per year from 2013.

MedImmune’s past successes include two innovative vaccines: the first monoclonal antibody approved by the FDA for use against an infectious disease (Synagis, used to prevent the childhood lung disease RSV); and the first intranasal vaccine against influenza (FluMist).

AstraZeneca has lost its appeal to the European Court of Justice against a €52.5m fine for its tactics to delay generic versions of Losec (omeprazole).

The company was fined by the European Commission in 2005 for manipulating regulatory approval in order to block generics.

According to the Commission, AZ launched a tablet version of the ulcer drug in 2001 and asked for the capsule’s authorisation to be withdrawn.

As a result, generic companies planning to launch their own capsule versions of omeprazole were blocked.

The Commission also claimed that AZ had deceived patent offices, courts and lawyers in several EU states over the date of Losec’s authorisation.

Losec, a proton pump inhibitor, was the world’s best-selling drug in 2000, but its European patent expired in 2001.

The European Commission commented that the appeal decision would prevent other companies from misusing drug regulatory procedures in order to protect their drug patents.

According to Ana Nicholls, Healthcare Analyst at the Economist Intelligence Unit, “This case exposed some of the strategems that pharma companies have used to delay the launch of cheaper generic versions of their drugs.

“By upholding the ruling, the court confirmed that pharma companies have to stamp out these practices or risk a substantial fine.”

Jonas Koponen of London law firm Linklaters commented that the decision was representative of “the European Commission’s policy of removing obstacles to competition from generics”.

AstraZeneca is to shed around 400 jobs in its German division as part of a corporate restructuring programme.

The company will integrate its Commercial Excellence and Strategic Planning divisions into other departments and reduce its workforce to around 625 personnel.

Gabriel Baertschi, Managing Director of AstraZeneca Germany, said the structural changes are “painful but necessary” to protect the future of the company and maintain a “competitive positioning in the market.”

AZ said the decision to cut jobs was made to adapt to a changing European economy and market pressures. It also points towards a changing product portfolio and delays in research and development of new drugs, as well as “massive state intervention” in the pricing of innovative medicines in Germany.

Staff were notified of the decision in a meeting with senior management yesterday. They were offered voluntary redundancy or early retirement. AZ said it would assist former employees to find a new job.

“With our settlement offer and the support in the search for a new job, AstraZeneca make the points reduction socially acceptable,” said Gabriel Baertschi.

The Medical Research Council (MRC) has committed £7m funding to support 15 academic drug research projects using compounds from AstraZeneca.

The collaborations are expected to improve our knowledge of ways to treat major diseases such as dementia and cancer, as well as rare diseases such as motor neurone disease.

AstraZeneca made 22 pharmaceutical compounds, which it had validated as suitable for further research, available to medical researchers in December 2011.

Eight of the research projects will involve clinical trials of new drug candidates, while the other seven will involve laboratory and animal models.

“This landmark collaboration will see our leading scientists working with industry to find new insights into disease,” added David Willetts, Minister for Universities and Science. “It will speed up the search for innovative treatments and keep the UK at the forefront of biomedical research, which will in turn drive growth and deliver benefits for patients.”

The MRC received 23 full funding proposals, which it assessed independently of AstraZeneca. The recipients include research teams at universities in Manchester, Leeds, Sheffield, London, Glasgow, Birmingham, Edinburgh and Bristol.

The announcement was welcomed by the BioIndustry Association (BIA), whose Chief Executive Steve Bates said: “Today’s funding announcement signals the beginning of an exciting coming together of academia, industry and government to create new pathways to develop novel therapies for a range of serious conditions.

“The prospect of de-risking private investment in this way and supporting the development of these products for patients through innovative ways of working shows how the biopharmaceutical sector is responding creatively to the challenge of getting more treatments to market.”

Incivo, Victrelis and Mepact win recognition at the 2012 UK Prix Galien Awards.

Two new medicines for the treatment of Hepatitis C have won the 2012 UK Prix Galien Innovative Product
Award. Incivo (Janssen) and Victrelis (MSD) fought of stiff competition to win the prestigious prize at London’s House of Commons. The chairman of the judging panel, Professor Sir Michael Rawlins, said the treatments provided a perfect example of how the pharmaceutical industry can “demonstrate and justify its place in healthcare by innovating for change and showing real gains to the world.”

The ceremony also saw Takeda become only the third winners of a Prix Galien Award for orphan drug development. Mepact – for the treatment of osteosarcoma, a rare malignant bone tumour – won the Orphan Drug Award.

UK Prix Galien 2012
The UK Prix Galien, organised and managed by the specialist market access consultancy WG Consulting – which owns the UK franchise – is held every two years. The 2012 awards were hosted by former shadow Minister for Health Kevin Barron MP, who was the event’s Parliamentary Sponsor. Barron, who is currently co-Chair of the Associate Parliamentary Health Group, said: “It’s a privilege to be able to witness, at first hand, just a glimpse of the deep volumes of medical innovations being developed here in the UK. As an MP, I’ve had a long-standing professional acquaintance with UK pharma. I know and recognise the many
benefits UK medicines have brought – and continue to bring – to patients all over the world. The sector’s continued commitment to the development of medicines to tackle disease, improve health outcomes and extend life is both remarkable and humbling.”

Barron said there was political consensus that driving improvements in health outcomes across all major diseases is a key priority for the NHS – and this focus had been reflected in the 2012 finalists. “It’s interesting to note that the shortlisted entrants for the 2012 UK Prix Galien show that pharmaceutical innovation is aligned with many of the priority needs identified in the NHS Outcomes Framework. Finalists include innovations for the treatment of diseases in cardiovascular, hepatology, mental health, neurology, gastroenterology and oncology. In addition, Prix Galien’s recognition of the industry’s attempts to treat rare, orphan diseases, once again underlines the very human value of R&D.”

Value-based message
The architect of the NHS Outcomes Framework, former Health Secretary Andrew Lansley, also addressed the audience. Attending his fourth consecutive UK Prix Galien, Lansley said: “Every time I come to this event I hear about fascinating innovations that I know are going to be at the heart of the health service for years to come. I’ve met – and continue to meet – patients that have benefited directly from innovations that I’ve previously heard about at Prix Galien. The HPV vaccination programme we have been able to roll out is just one example of that. So it’s a privilege to be here.”

Lansley said that recognising and rewarding innovation is a key Government priority – and that the publication of Innovation Health and Wealth last December was part of a consistent value-based message
it wanted to send to the NHS. “That message is that as you, the pharmaceutical industry, bring forward new treatments that will clearly add value and improve the quality of healthcare for patients then the NHS should be at the forefront, internationally, of demonstrating that value. Our health service can be an exemplar and inspiration to people around the world because of its capacity to demonstrate the effectiveness of new treatments when they are used within the NHS.”

Lansley praised the UK pharma industry, highlighting the value its innovations bring both to the economy and to patients worldwide. “What you are doing is part of how this country will pay its way in the future,” he said. “And it has the added value of knowing that, in the process, we can give patients in this country access to the very best healthcare anywhere in the world.”

The recognition of innovation that can lead to improved health outcomes is a core aim of Prix Galien, as outlined by Professor Sir Michael Rawlins, who announced the winners. “Prix Galien is about honouring excellence in pharmaceutical research and development,” said Professor Sir Michael. “It is about recognising the contribution that new medicines can make to the lives of people with life-threatening conditions. It is about celebrating the achievements of all those individuals – working as teams – upon whom we rely for the discovery and development of new medicines. Most will be unknown to us – but we all owe them a huge debt of gratitude.”

Innovative Product Award
The prestigious Prix Galien medal for innovation was jointly awarded to Janssen and MSD for their respective hepatitis C treatments Incivo and Victrelis. In the UK, it is estimated that there are between 200,000 and 400,000 people chronically infected with hepatitis C virus. This may lead to liver cancer as well as other serious liver diseases. Infection with the hepatitis C virus poses a substantial global health burden, and is responsible for 40% of all cases of end-stage cirrhosis, 60% of hepatocellular carcinoma and 30% of liver transplants.

Professor Sir Michael Rawlins said: “Hepatitis C virus has become an enormous area of need globally, with many patients unaware that they are infected. The consequences of this virus are considerable and burdensome to both patients and the healthcare system; current treatments remain ineffective in a significant number of cases whilst being unpleasant and poorly tolerated by patients themselves.

“Hepatitis C infection is a perfect example of where the pharmaceutical industry can demonstrate and justify its place in healthcare by innovating for change and showing real gains to the world. It is for this reason that the panel felt that both Janssen and MSD should be celebrated and congratulated for their part in addressing the ongoing challenge in managing HCV and its associated complications.”

Brilique (AZ) and Resolor (Shire) both received commendations. Gilenya (Novartis), Xarelto (Bayer), Xeplion (Janssen), Xgeva (Amgen), Yervoy (Bristol-Myers Squibb), Zelboraf (Roche) and Zytiga (Janssen) were all shortlisted.

Orphan Drug Award
The Orphan Drug Award was introduced as a dedicated category at 2008 UK Prix Galien. There had previously been a special award for orphan products in 2006. The term ‘orphan condition’ is used to describe conditions that affect a very small number of patients in a given population – many of which are either untreatable or treated very inadequately. It is estimated that there are 6,000 orphan diseases – which, in total, affect about 30 million EU citizens.

“For orphan diseases that are potentially treatable with medicines, pharmaceutical manufacturers face a number of hurdles – including concerns about the size of the market and difficulties because of the small numbers of patients – in their development,” said Professor Sir Michael.

The 2012 Orphan Drug Award was won by Mepact from Takeda. Mepact (mifamurtide) is for the treatment of osteosarcoma, a rare malignant bone tumour – mainly of children and adolescents – that affects fewer than 1 per 10,000 individuals in the EU. This is equivalent to 150 children and young adults each year in the UK. Tumours most frequently occur in the long bones and are highly aggressive with a propensity to metastasise, particularly to the lung. If left untreated, the primary tumour will undergo local and systemic progression, leading to death within months.

“To investigate the role of this immune modulator in osteosarcoma required extensive and complex trial design with careful implementation of the study programme,” said Professor Sir Michael. “Apart from its novel mechanism of action – and clear evidence of its clinical effectiveness – the jury were also extremely impressed that such an advance in the management of osteosarcoma represents the first significant change in outcomes in 10–20 years of managing this disease. That Takeda managed to undertake the clinical development of this product – in such a niche indication – is hugely to their credit.”

A host of pharmaceutical and biotechnology companies will join forces alongside public research organisations and scientific experts to address the rising threat from antibiotic resistance.

Companies including GSK, AstraZeneca, Janssen and Sanofi will partner in a new £180 million research programme to boost the discovery and development of new antibiotics.

Patrick Vallance, President, Pharmaceuticals R&D at GSK said the agreement “signals a new model of collaboration” to develop treatments to tackle infections such as MRSA.

NewDrugs4BadBugs is supported by the Innovative Medicines Initiative (IMI) – who will jointly fund the first project with contributions from the pharma and biotech companies involved.

The objective of the collaboration is to improve the underlying scientific understanding of antibiotic resistance and design and implement clinical trials that see novel drug candidates clinically developed.

Currently, the WHO describes the pipeline of future antibiotics to counter emerging resistant bacteria as “virtually dry”.

Antibiotic resistance is increasingly becoming a worldwide health threat with new ‘superbugs’ evolving around the globe.

However, despite the urgent need for effective counteraction, research has diminished over the past 15 years with few companies remaining active in this area due to difficulties in finding new agents and regulatory complexities.

Martin Mackay, President, R&D, at AstraZeneca commented: “It is time to tackle this issue in a different way, sharing information and expertise among public and private partners – collaboration of this type is critical if we are to speed up the discovery of these medicines to improve patient health.”

AstraZeneca looks set for several agreements to boost its pipeline after suffering a host of late-stage setbacks, according to Dr Martin Mackay (pictured), Head of Research and Development.

The R&D chief indicated the pharmaceutical company is hoping to sign deals with other major pharma companies as well as targeting biotech acquisitions and licensing partnerships.

Speaking to the Financial Times, Dr Mackay said he would be “personally disappointed” if agreements to secure AZ’s future were not completed by the end of the year.

The company has been linked as one of the suitors interested in acquiring Amylin Pharmaceuticals, who reportedly rejected a hostile bid from BMS.

Shareholders at the Anglo-Swedish pharma firm have criticised the leadership of retiring CEO David Brennan for failing to securing merger and acquisition deals to compensate for its failing pipeline.

Mr Brennan announced his departure on the day AZ published their Q1 financial results which revealed an 11% drop in revenue after key brands lost patent protection.

The outgoing CEO said the pharmaceutical industry is “experiencing pressures none of which I’ve witnessed in my 36 years in the industry” but remains “very confident” AZ has the “capabilities, courage and determination to be successful into the future”.

Dr Mackay insists his research teams have remained “undistracted by recent events” at the company and said the department continue to do “the right things”.

Since he joined AZ from Pfizer in 2010, the R&D head has restructured the department, outsourcing several in-house activities whilst axing 40% of its investigational portfolio.

GSK Chief Executive Sir Andrew Witty has ruled out a takeover bid for AstraZeneca.

Sir Andrew told shareholders at the company’s annual general meeting yesterday not to expect any major takeovers in the future as GSK focuses on the potential of its pipeline.

In response to a question over a possible merger, Sir Andrew said a deal for AZ would be “very distracting” at a time when experimental drugs in its pipeline are entering an exciting period.

The future of AstraZeneca remains uncertain as the company battles against generic competition, setbacks in drug development and the loss of its CEO David Brennan after he retires on June 1.

Revenue was down by 8% in the first three months of this year at AZ with sales in the US, Western Europe, Established Rest of the World and Emerging Rest of the World all falling.

David Brennan announced his retirement to coincide with the publication of the Q1 results after admitting that the pharmaceutical sector is “experiencing pressures none of which I’ve witnessed in my 36 years in the industry”.

AZ shareholders had criticised his leadership in recent years after the company had failed to compensate for the loss of revenue with mergers and acquisitions.

As a result, industry analysts have speculated that AZ may become a takeover target for one of pharma’s biggest companies.

A merger of GSK and AZ, the two largest pharma companies in the UK, would provide big cost savings. It led one GSK shareholder to raise the issue with the Chief Executive claiming it would be more effective than the recent $2.6bn offer for Human Genome Sciences.

Sir Andrew said GSK believes it can “deliver an extraordinary return to shareholders through this acquisition. I think we waited until exactly the right moment to make this offer, but nonetheless this is a compelling offer for shareholders at HGS to consider,” he said.

The $13 per share offer was rejected by HGS – who have now instructed Goldman Sachs and Credit Suisse to help explore strategic alternatives to GSK’s bid. Sir Andrew declined to comment on whether he had made contact with HGS’ management since GSK’s offer was rejected.

Novartis is expected to overtake Pfizer and become the biggest manufacturer of prescription medicines by 2018, according to new consensus data.

Research by EvaluatePharma estimates Novartis will record sales of more than $50bn in six years’ time, with its eye care business Alcon and generic unit Sandoz driving growth.

But the outlook is not good for US-based companies with only Pfizer remaining in the top five by 2018 and Sanofi, GSK and Roche maintaining a strong presence.

Data found that despite generic competition on Diovan and Glivec and disappointment from key projects such as Gilenya and its new respiratory franchise, Novartis is expected to record annual growth between 2011 and 2018 of 1.2%.

This is in contrast with AstraZeneca whose annual sales are expected to drop from $32.4bn in 2011 to $22.1bn in 2018 representing a negative growth of 5.3%.

Gilead Sciences is expected to experience the biggest increase in annual growth of the top fifteen companies with data showing sales will rise from $8.1bn to $15.7bn at a rate of 9.9% per year.

Novo Nordisk is also forecast to enter the top 15 ranked companies for the first time due to an increasing demand for its diabetes medicines. Annual growth is expected to be 7% until 2018 with sales totalling nearly $20bn.

One of the biggest casualties, data found, will be Eli Lilly. The Indianapolis research-based company currently claims to be the 10th biggest pharmaceutical company in the world. But Lilly fails to make the top 15 companies after research found a drop in sales will see it fall to 17th place by 2018. But researchers did note that Lilly’s Alzheimer’s candidate, solanezumab, could reverse the trend if it successfully enters the lucrative market.

Lilly will be replaced in the list by German healthcare giant Bayer, which also enters the top 15 global companies for the first time, with annual sales of around $16.5bn by 2018 boosted by Xarelto.