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.”

The FDA has said that MS drug Gilenya (fingolimod) should not be used in patients with a recent history of stroke or heart disease.

The requested label change adds to changes requested by the EMA and the FDA in April to reduce the risk of cardiovascular and neurovascular events.

The FDA also concluded that a number of sudden deaths in patients with severe MS taking Gilenya could not be conclusively linked to the drug.

Gilenya was recommended by NICE in late April as a “valuable new therapy” for severe relapsing-remitting MS.

The new FDA safety review reinforced the earlier recommendation that doctors monitor the heart rate of patients taking Gilenya for the first time.

However, it said that the death of a 59-year-old patient in November after a first dose of Gilenya could have been caused by the patient’s advanced brainstem MS lesions and not by the drug.

In other cases of sudden death in patients taking Gilenya, it said, the drug’s “contribution to the death was unclear”.

However, the FDA definitely recommended not using Gilenya in patients who have suffered a stroke or heart disease within the previous six months, or who are taking certain medications for cardiac arrhythmia.

Gilenya was approved in the US in 2010 and in the EU in 2011, and remains the only oral MS drug on the market.

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.

Novartis has reported an 18% drop in its profits for the first quarter of 2012, due to generic competition and manufacturing problems.

The Swiss company’s net profit fell to $2.33bn from $2.82bn in the same period last year, while its sales revenue fell by 2%.

“We expected a challenging quarter in Q1, and we delivered in line with our expectations,” said Novartis CEO Joe Jiminez.

He highlighted the impact of the temporary closure of the company’s factory in Nebraska, USA, which cost Novartis £200m in quarter 1.

Novartis’ generics business suffered from further production problems, which combined with growing competition to reduce its sales by 10%.

Jimenez said the company would restart production at its Nebraska site, but expected its full-year profits to be below the 2011 figure.

However, he said that Novartis expected sales of over $1bn this year from its nine leading drugs, including Galvus (for diabetes), Tasigna (for leukaemia) and Lucentis (for wet AMD).

In addition, he said, the company’s oral MS drug Gilenya was set to achieve ‘blockbuster’ status, despite safety concerns that have led to label changes.

NICE has reversed its original decision on Novartis’ Gilenya (fingolimod), the first pill to treat multiple sclerosis, in final draft guidance.

Gilenya is now recommended after NICE assessed a public consultation which Novartis and clinicians provided additional information and analysis on the effectiveness of the medication.

Sue Webb, General Manager, UK & Ireland and Country President, Novartis UK, said the treatment “will make a real difference” to people living with MS.

The recommendation follows two previously failed appraisals. After the first in August 2011, Novartis submitted a Patient Access Scheme (PAS) to provide a discount to the NHS.

But, following another consultation, NICE said the MS pill still did not represent value for money in a separate draft guidance document last December.

However, during the second consultation period, Novartis revised its analysis for a subgroup of patients for the licensed population. Following this, NICE’s independent Appraisal Committee now believes the treatment offers value for money to the NHS, when provided under the terms of the PAS.

The subgroup of patients includes adults with highly active relapsing-remitting multiple sclerosis, whose relapses have increased or remained compared to the previous year, despite the use of beta interferons.

NICE now provisionally recommends the treatment for adults who have an unchanged or increased relapse rate or ongoing relapses compared to the past year, despite treatment.

Dr Eli Silber, a consultant neurologist at King’s College Hospital, said she was delighted NICE reversed its original decision. “We’ve waited a long time for an effective oral treatment to offer patients who are continuing to relapse on first line injections,” said Dr Silber. “Today’s decision increases treatment choice. Because it is a highly effective oral agent it may change the way MS is managed in the UK forever.

“With more active forms of MS, we have a limited window of opportunity to make a difference to patients’ lives – many are young people who are raising families and starting their careers. I want to get appropriate patients onto this therapy as quickly as possible.”

NICE has again been unable to recommend the first pill licensed in the UK for highly active relapsing remitting multiple sclerosis (RRMS) in further draft guidance.

It claims that Gilenya (fingolimod) is not an effective use of NHS resources when compared with existing treatments – despite an improved Patient Access Scheme by Novartis.

Sir Andrew Dillon accepted the clinical benefits of the pill but said NICE “has not been convinced that it is a cost effective treatment option for the NHS”.

The second draft guidance has been published following a proposed discount from Novartis to the price of Gilenya, which it claims to have made “every effort to make this first oral therapy affordable”.

The pharma company says NICE has ignored “overwhelming support from the MS community and general public” in making the decision and questioned the difference of opinion regarding clinical practice in the UK and raised concerns about the impact of the decision and length of review process for patients.

Novartis disagrees with the emphasis NICE placed on the best supportive care when comparing Gilenya and has now called on the MS community to appeal against the decision during the consultation period which is open until early January. NICE’s Appraisal Committee will then meet again in February to review the comments with final guidance expected to published in April 2012. 

Sanofi is expected to overthrow Pfizer’s nine-year reign as the world’s biggest drug maker, according to new research.

The French pharmaceutical company is expected to retain the top spot until at least 2016, with Pfizer falling to third place behind Novartis due to the loss of Lipitor’s US patent protection, according to EvaluatePharma (see figure one).

The report says that Sanofi’s numerous acquisitions over the last decade have contributed largely to the company’s success, gaining $20 billion after it bought out Genzyme.

Sanofi’s mergers over the last decade have contributed a great deal to its current position, starting with its $30 billion deal with Synthélabo in 1999.

It is expected that the company will retain its top position until at least 2016, mainly thanks to sales of enzyme replacement therapies through its acquisition of Genzyme.

Also, the company’s addition of Cerezyme and Myozyme blockbuster drugs will help fill the gap left by Lovenox, Taxotere, and Plavix, which loses US patent protection in 2012.

Pfizer’s $68 billion buyout of Wyeth in 2009 helped fill the gap left by Lipitor, but it will be difficult to replace the drug’s global sales figure of $13.4 billion seen in 2008, which set the record as the biggest selling medicine.

Following its loss of US patent protection in November 2011, Lipitor sales are estimated to shrink to $2 billion by 2016.

However, pipeline products such as rheumatoid arthritis (RA) pills tofacitinib and Eliquis are expected to boost Pfizer’s drug sales after 2016, which will help retain the company’s position in the top-five pharmaceutical companies.

Merck’s four-year outlook is seen as bleak despite its takeover of Schering-Plough for $41 billion in 2009, with only 1% annual sales growth predicted, conceding to European companies GlaxoSmithKline and Roche to overtake the company.

EvaluatePharma predicts that Johnson & Johnson’s recent pipeline successes will benefit the company in the coming years, despite its drugs arm being substantially smaller than the five biggest pharma companies.

It is thought that Novartis will be Sanofi’s closest competition over the next few years, with strong sales growth from Gilenya and Tasigna due to Diovan’s loss of patent protection next year.

Figure 1:

Novartis will cut 2,000 manufacturing jobs in Switzerland and the US – despite seeing an increase in group net sales and operating income in Q3.

The positions will be cut by 2016 after the closure of two Swiss production sites and one in Italy, plus the restructuring and relocation of R&D activities from Switzerland to the US.

Joseph Jimenez, CEO of Novartis, said the job losses are needed for the Group to “continue delivering on our mission of bringing innovative new drugs to patients.”

However, the job losses will be offset by the creation of 700 new positions in low cost and other countries, the company said.

The Group recorded a sales increase of 12% in constant currencies to $14.8 billion and saw income also grow by 11% to $4.1bn.

Sales were boosted by a strong performance from recently launched products – which contributed 25% of net sales.

Pharmaceutical sales grew 9% after Afinitor (pictured)/Votubia was approved in the EU for two additional indications, the CHMP granted a positive opinion for Rasitrio for high blood pressure and the company’s breakthrough multiple sclerosis treatment, Gilenya, won approval in Japan.

“Once again, the breadth of our business and product portfolio allowed us to deliver strong financial results and operating leverage, as well as significantly advancing the pipeline in the quarter,” said Mr Jimenez. “To strengthen our future, we have accelerated actions to reduce our cost base over the next few years.”

NICE has not recommended the use of Novartis’ Gilenya (fingolimod) for the treatment of a particular type of multiple sclerosis in provisional draft guidance.

Questions were raised by NICE’s independent Appraisal Committee over uncertainties of the drug’s clinical effectiveness based on Novartis’ evidence.

Professor Carole Longson, Director of the Health Technology Evaluation Centre, says Novartis did not provide “sufficient evidence” to show the treatment is better than other recommended options.

The Institute says that clinical trials have shown that Gilenya can reduce the number of relapses in certain people with RRMS. But it is unclear how much the treatment may help the specific group of people for whom it is licensed.

Gilenya is approved for adults who have highly active relapsing-remitting multiple sclerosis (RRMS) and experience at least one relapse a year, despite being treated with beta interferon, and for those with rapidly evolving severe RRMS who experience two or more relapses regardless of their treatment.

Novartis submitted evidence which focused solely on a sub-group of patients with rapidly evolving severe RRMS. The data also compared Gilenya with a placebo, and with a specific type of beta interferon that is not widely prescribed in the NHS.

Because of this the independent Appraisal Committee were unable to determine how much Gilenya reduces the rate of relapses compared with other recommended treatments. The Committee says that Novartis should’ve submitted a comparison with other beta interferons, and with the recommended Tysabri (natalizumab).

“While it’s important that people with multiple sclerosis have treatment options, NICE has to ensure that the NHS provides treatments that bring benefits that are value for money,” Dr Longson said. “This is so that everyone who uses the NHS can receive the best care possible.

“Based on the available clinical evidence and economic analysis, our independent committee concluded that fingolimod would not be effective good use of NHS resources.”

The draft guidance is now open to comment with final guidance expected in December.

Novartis posted an increase in profits of 12% to $2.73 billion in Q2 of 2011 after strong sales of new products and growth in emerging markets.

Net sales grew by 27% to $14.9 billion after the recent launch of multiple sclerosis medicine Gilenya, and strong results from its generic drug division Sandoz, and eyecare unit Alcon.

Joseph Jimenez, CEO, Novartis, says the company’s diversified healthcare portfolio is “enabling us to generate superior results”.

The Swiss-based company also released four new products during the second-quarter of this year.

The FDA approved both Afinitor for advanced pancreatic neuroendocrine tumours and Arcapta Neohaler for COPD; whilst the EU granted its approval for Lucentis to treat retinal vein occlusion, and hypertension medicine Rasilamlo.

The FDA also accepted an application to expand Menveo’s indication to toddlers and infants as young as two months, with an additional application filed in Europe for the Janus kinase inhibitor INC424 to myelofibrosis.

Mr Jimenez said the company’s “excellent execution behind a sound strategy” has resulted in another successful quarter.

“We achieved strong sales growth of 19% in constant currencies and margin improvement of 0.4 percentage points in US dollars. We further demonstrated the success of our R&D strategy with four major approvals and two filings in the second quarter.”